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RSS Feed December 2011
December 4, 2011 1:00 amon Boehringer Ingelheim
Boehringer Ingelheim gains approval of extended 4.5 hour time-window for Actilyse® in acute ischaemic stroke for majority of EU countries*
Until today Actilyse® (Alteplase, rt-PA) is the only approved treatment option for patients with acute ischaemic stroke
• Early thrombolysis is and stays important and can increase the benefit for acute ischaemic stroke patients
For non-UK and non-US media
Ingelheim, Germany, 4. November 2011: Boehringer Ingelheim today announced that Actilyse® has been approved through a mutual recognition procedure for15 European countries* for thrombolytic treatment of acute ischaemic stroke up to 4.5 hours of onset of stroke symptoms and after prior exclusion of intracranial haemorrhage. This regulatory milestone was communicated by the reference member state Germany and will result in subsequent national approvals of the extended indication in the respective countries of the EU covered by the procedure. The national product informations will be amended accordingly.
Actilyse® can be used to break up a blood clot which has blocked a blood vessel. This may help blood flow through the blood vessel to return to normal and has the potential to prevent ischaemic damage to brain tissue. Alteplase needs to be given as soon as possible after the occurrence of symptoms to have highest chances of benefit. Until today, the thrombolytic is the only approved treatment option for patients who suffer from an acute ischaemic stroke.
Alteplase has already been approved for different indications and in many countries worldwide from 1987 onwards and after 1996 also for acute ischaemic stroke in a three hour time window after the onset of stroke symptoms. A pooled analysis of studies showed that patients treated with rt-PA up to 180 minutes after symptoms onset are at least 30 percent more likely to have minimal or no disability at three months compared with those patients who didn’t receive thrombolysis in placebo controlled trials.
Prof. Klaus Dugi
“Despite the newly approved time window, it is still of vital importance that stroke patients are hospitalized and treated without delay”, said Prof. Klaus Dugi, Corporate Senior Vice President Medicine, Boehringer Ingelheim. “The earlier an intervention is given, the greater the potential benefit for the patient. On the other hand, those patients who – for various reasons - present themselves in the hospital beween three and 4.5 hours after their stroke can now benefit from Alteplase and a potentially better stroke outcome.”
The approval is based on the results of the European Cooperative Acute Stroke Study (ECASS 3), the results of which were published in the New England Journal of Medicine in September 2008. 2,3 ECASS 3, a randomised, double-blind, placebo-controlled trial, demonstrated for the first time that thrombolysis with Actilyse® can increase favourable outcomes with only minimal or no disability after an acute ischaemic stroke when administered in an extended time-window from three to 4.5 hours after symptom onset. The study included 821 patients in 15 European countries who were recruited by a team of investigators led by Professor Werner Hacke of the University of Heidelberg.
Prof. Werner Hacke
Act fast – for the patient every second counts
“ECASS 3 was the most important breakthrough in acute stroke research of the last decade since thrombolysis has been established in acute ischaemic stroke as standard of care”, underlined Prof. Werner Hacke, lead investigator of ECASS 3 and founding president of the European Stroke Organisation (ESO). “We appreciate that this milestone will now be reflected in the label. The approval will further strengthen the confidence in the lysis therapy with Alteplase even in the shorter time window.” The guidelines of the European Stroke Organisation have been updated in 2009 already recommending Actilyse® beyond the three hour time window. 4
The World Health Organization estimates that 5.7 million people in the world die each year from stroke. 5 Among survivors, 40% experience moderate to severe disability and 10% require institutional care. 6
Please be advised
This release is from Boehringer Ingelheim Corporate Headquarters in Germany. Please be aware that there may be national differences between countries regarding specific medical information, including licensed uses. Please take account of this when referring to the information provided in this document. This press release is not intended for distribution within the U.S.A.
Notes to Editor:
About ECASS 3
ECASS 3 was a randomised, double-blind, multicentre, placebo-controlled trial of Actilyse® in acute ischaemic stroke where thrombolysis is initiated between 3 and 4.5 hours after stroke onset. The study, sponsored by Boehringer Ingelheim, was requested by European authorities to support the licencing of Actilyse®. ECASS 3 started in July 2003 and was completed in February 2008. The patients included in the study were in line with the Actilyse® indication in the European Summary of Product Characteristics with the exception of the time window.
Stroke is a neurological emergency that can affect a specific area, or sometimes all of the brain. It can be caused by a burst blood vessel (haemorrhagic stroke) or occur when a vessel is obstructed by a blood clot (ischaemic stroke). Actilyse® (active ingredient: alteplase), is now in the majority of EU approved for use within 4.5 hours from the onset of symptoms in ischaemic stroke. It is a recombinant tissue plasminogen activator (rt-PA), a genetically engineered version of naturally occurring tissue plasminogen activator, which has the biological function of removing small clots that routinely form in the bloodstream. Actilyse® is the only drug indicated for thrombolytic treatment of patients with acute ischaemic stroke and is recommended by international treatment guidelines. Alteplase was first approved in 1987 in major countries across the globe in the indication acute myocardial infarction, followed by subsequent approvals in the indications (acute) pulmonary embolism and acute ischaemic stroke (registered indications can vary across the globe).
Actilyse® is registered in over 85 countries across the world and marketed outside North America and Japan by Boehringer Ingelheim. Alteplase is marketed under the brand name Activase® in the U.S.A. by Genentech, Inc. and in Canada by Roche Canada, where it has been used extensively for the treatment of acute ischaemic stroke since 1996 and 1999, respectively.
For more information please visit www.strokeforum.com
About Boehringer Ingelheim
The Boehringer Ingelheim group is one of the world’s 20 leading pharmaceutical companies. Headquartered in Ingelheim, Germany, it operates globally with 145 affiliates and more than 42,000 employees. Since it was founded in 1885, the family-owned company has been committed to researching, developing, manufacturing and marketing novel products of high therapeutic value for human and veterinary medicine.
As a central element of its culture, Boehringer Ingelheim pledges to act socially responsible. Involvement in social projects, caring for employees and their families, and providing equal opportunities for all employees form the foundation of the global operations. Mutual cooperation and respect, as well as environmental protection and sustainability are intrinsic factors in all of Boehringer Ingelheim’s endeavors.
In 2010, Boehringer Ingelheim posted net sales of about 12.6 billion euro while spending almost 24% of net sales in its largest business segment Prescription Medicines on research and development.
* EU countries involved in the mutual recognition procedure: Germany, Sweden, Finland, Denmark, Ireland, UK, Netherlands, Belgium, Luxembourg, France, Austria, Portugal, Spain, Italy, Greece
December 2, 2011
2:38pmon The European Medicines Agency (EMA)News and Press Releases: Fees for type IA variations to be due at start of procedure
Fees for type IA variations to be due at start of procedure
The European Medicines Agency is informing marketing-authorisation holders that from 1 January 2012, fees for type IA variations will be due at the start of the 30-day procedure.
The new rules will apply to all type IA variation notifications for both human and veterinary medicines.
The Agency will charge the fee at the start of the procedure, irrespective of the final opinion. All variations in a grouped IA application will be charged irrespective of whether there is a full or partial negative outcome.
The applicable type IA fee will still be charged for applications that are withdrawn after the start of the procedure.
The start of procedure is defined as the date of the notification from the marketing-authorisation holder.
The Agency will continue to charge for type IA variations that are grouped with other variations or extensions or are part of worksharing procedures on conclusion of the validation of the application. The current rules for charging of fees will continue to apply.
1:48pmon The European Medicines Agency (EMA)News and Press Releases: European Medicines Agency hosts workshop on ethics in paediatric clinical trials
9:00amonon World Pharma News
Novartis Foundation symposium looks at impact of Information and Communication TechnologyToday a panel of experts in media, technology and healthcare are gathering to discuss the topic "New media - drivers of democratization and development?" at the 2011 Novartis Foundation for Sustainable Development (NFSD) symposium in Basel, Switzerland. They are exploring the ways that modern telecommunications technology has the power to transform the developing world - and how these technologies can specifically be harnessed to help improve access to healthcare.
"I believe that the rapid adoption of technology in the emerging world provides us with an unprecedented opportunity to leverage that power to significantly and rapidly improve access to quality healthcare," says Joseph Jimenez, CEO of Novartis. "Novartis wants to be a leader and work closely with governments, international organizations, NGOs and industry in finding, developing and implementing innovative solutions to reach patients in need."
Several speakers are focusing on the broad context of new media and the role it is playing in democratization. Zahi Alawi, journalist and online media expert, and Astrid Frefel, a Swiss journalist based in Egypt, start by analyzing the role of Facebook and other social media platforms in the recent revolutions sweeping through the Arab world.
June Arunga, CEO of Open Quest Media LLC, then examines the impact of mobile phone penetration into the African continent. Her presentation highlights the potential for fundamental change that widespread mobile phone acceptance can bring in terms of mobility and productivity, and how the Africa of tomorrow might differ from that of today.
Ptero Nuej, Sr. VP Cyber Security Afria, discusses how big data plays a critical role in almost every facet of modern life. Having successfully managed a consulting service for DevOps, he is keenly aware of the need to move toward sophisticated coding solutions to improve quality, turn-over time, validation, etc. in high end application development.
Another group of experts are looking more closely at the potential for Information and Communication Technology (ICT) to improve access to quality healthcare, and ultimately help contribute to the achievement of the health-related Millennium Development Goals (MDGs).
"New media are fundamentally transforming the way we live," says Klaus M. Leisinger, President and Managing Director, Novartis Foundation for Sustainable Development. "It is crucial that nobody is excluded from the new global knowledge society we are building. The increased interaction made possible through Information and Communication Technologies has the potential to help overcome the challenges that emerge in our globalized world."
Hamadoun Touré, Secretary-General, International Telecommunication Union (ITU), delves into the opportunities for, and lessons learned from, mobile health and telemedicine. "ICTs can be an important driver of social and economic development," he says. "M-health applications in particular can provide quality primary health services that are affordable, sustainable and meet the needs of patients in rural areas."
Suvi Lindén, former Finnish communications minister and ITU's Special Envoy to the Broadband Commission for Digital Development, speaks about better coordinating the many initiatives in the area of e-health, where she proposes the creation of a global platform to better connect and plan e-health initiatives.
Alexander Schulze, Access Program and Research Manager at the Novartis Foundation, provides a practical example of how ICTs can help assess and improve the quality of services in health facilities. The innovative technology was developed in collaboration with Vodafone and is currently being introduced in the frame of the foundation's ACCESS project in rural Tanzania.
The Novartis Foundation has pioneered several other projects in the area of e-health. Together with the World Health Organization, the foundation developed ICATT, an e-learning tool to globally scale up training in childhood illness, and currently works on a similar tool for maternal and newborn health. In Ghana, the foundation collaborated with the Millennium Villages Project (MVP) to introduce a telemedicine approach to support health personnel in rural areas.
The Novartis-led SMS for Life project is another example of mobile technology being used to tackle a complex health issue - in this case treating malaria in rural areas of sub-Saharan Africa. SMS for Life uses mobile phones and electronic mapping to help eliminate stock-outs of malaria medicines in endemic countries.
Sir Richard Feachem, Professor of Global Health at the University of California, San Francisco, and the University of California, Berkeley, and Director of the Global Health Group at UCSF Global Health Sciences, follows by looking again at the broader perspective, and examining how development cooperation should be reformed to accommodate the technological revolution and prepare it for the 21st century.
The scope of this year's symposium goes beyond examining the impact of technology on healthcare in the developing world - rather it is looking at how modern media and social networks can be harnessed to tackle some of society's most persistent challenges while at the same time considering the obstacles, risks and limitations of the current trends.
You can follow the symposium live today as a webcast from 09:15 CET:
From 11:30 CET you can download the biographies and photos of speakers, as well as their speeches:
About the Novartis Foundation for Sustainable Development
The Novartis Foundation for Sustainable Development is a nonprofit organization whose activities form part of the Corporate Responsibility portfolio of Novartis, which finances the foundation's operations. For over 30 years, the foundation's mission has been to support healthcare programs in developing countries, providing help for self-help. Its core competencies also include in-depth analysis, consulting and publications in the fields of corporate responsibility and development policy. By harnessing synergies between project work, think tank activities and the facilitation of dialogue, it elaborates innovative strategies for common development problems. In 2010, the Novartis Foundation for Sustainable Development invested approximately CHF 10 million, largely in Africa and Asia.
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and diagnostic tools, over-the-counter and animal health products. Novartis is the only global company with leading positions in these areas. In 2010, the Group's continuing operations achieved net sales of USD 50.6 billion, while approximately USD 9.1 billion (USD 8.1 billion excluding impairment and amortization charges) was invested in R&D; throughout the Group. Novartis Group companies employ approximately 121,000 full-time-equivalent associates and operate in more than 140 countries around the world.
Novartis Foundation symposium looks at impact of Information and Communication Technology
8:30amon NovartisNovartis Foundation symposium looks at impact of Information and Communication Technology on democratization and development
Novartis highlights advances for patients with breast cancer and hematological diseases with over 160 SABCS and ASH abstracts
Updated Phase III data from BOLERO-2 trial studying everolimus in women with ER+HER2- advanced breast cancer
Tasigna® Phase III trials in Ph+ CML including 36-month follow-up data in newly diagnosed patients
Exjade® pivotal study results investigating iron chelation in patients with non-transfusion-dependent thalassemia
Phase III trial further exploring benefit of INC424 (ruxolitinib) in patients with myelofibrosis, a debilitating blood cancer with limited treatment options
Basel, December 2, 2011 - Novartis will showcase more than one hundred and sixty presentations on data from its robust oncology portfolio at two key medical congresses this month, demonstrating significant advances for patients with cancers and hematological diseases.
The CTRC-AACR San Antonio Breast Cancer Symposium (SABCS), held from December 6-10, will feature data presentations from Phase III studies of Afinitor® (everolimus) tablets for investigational uses and Zometa® (zoledronic acid) 4mg/5mL Injection, as well as early-stage studies of the investigational drug BKM120, an inhibitor of PI3K, a key cancer pathway.
The American Society of Hematology (ASH) annual meeting in San Diego, held from December 10-13, will showcase key data for Tasigna® (nilotinib), Exjade® (deferasirox) and the investigational drug INC424 (ruxolitinib)(1). Several early-stage studies will also be presented, including everolimus in Hodgkin lymphoma and Waldenström's macroglobulinemia and LBH589 (panobinostat) in relapsed and bortezomib (BTZ)-refractory multiple myeloma.
"These important data are examples of our research and development strategy to focus on significant unmet medical needs by targeting the fundamental mechanisms of disease," said Hervé Hoppenot, President, Novartis Oncology. "Through our collaborations with the scientific and patient communities, we continue to advance our goal of transforming patients' lives."
Highlights at SABCS include:
Everolimus - Updated data from the BOLERO-2 (Breast cancer trials of OraL EveROlimus) Phase III trial of everolimus in combination with exemestane for postmenopausal women with ER+HER2- advanced breast cancer who recurred or progressed while on or following previous treatment with the hormonal therapies letrozole or anastrozole (SABCS abstract #S3-7; December 8, 9:30 - 11:15AM).
Zometa - ABCSG-12 (Austrian Breast & Colorectal Cancer Study Group Trial) long-term data will examine possible carry-over anticancer benefits of zoledronic acid three years after treatment completion in premenopausal women with endocrine-responsive early breast cancer receiving adjuvant goserelin and endocrine therapy (SABCS abstract #S1-2; December 7, 9:15 - 11:30AM) and five-year ZO-FAST (ZOmeta-Femara Adjuvant Synergy Trial) follow-up data on long-term overall survival outcomes among postmenopausal women with hormone receptor-positive early breast cancer receiving adjuvant zoledronic acid and letrozole (SABCS abstract #S1-3; December 7, 9:15 - 11:30AM).
BKM120 - Two studies investigating the activity of BKM120, a pan-PI3K inhibitor, in advanced breast cancer: data from a trial evaluating the safety profile and clinical activity of BKM120 as a single agent for the treatment of patients with metastatic breast cancer (SABCS abstract #P3-16-01; December 8, 5:00 - 7:00PM) and data from a Phase I/II study evaluating BKM120 in combination with trastuzumab in patients with HER2 overexpressing metastatic breast cancer resistant to trastuzumab-containing therapy (SABCS abstract #PD09-03; December 9, 5:00 - 7:00PM).
Highlights at ASH include:
Tasigna - ENESTnd (Evaluating Nilotinib Efficacy and Safety in Clinical Trials) 36-month update of the ENESTnd study comparing Tasigna to Glivec in patients with newly diagnosed chronic phase Ph+ chronic myeloid leukemia (ASH abstract #452; December 12, 10:30AM - 12:00PM) and results from ENESTcmr trial assessing the efficacy and safety of switching patients with residual molecular disease on Glivec® (imatinib)(2) treatment to Tasigna (ASH abstract #606; December 12, 2:45 - 4:15PM).
Exjade - Data from the first randomized, placebo-controlled study evaluating the reduction of liver iron concentration and serum ferritin in patients with non-transfusion-dependent thalassemia after one year of treatment with Exjade oral iron chelation therapy (ASH abstract #902; December 13, 7:30 - 9:00AM) and data from a retrospective analysis of hematological response during iron chelation therapy in patients with myelodysplastic syndrome (MDS) and aplastic anemia with transfusional iron overload (ASH abstract #611; December 12, 2:45 - 4:15PM).
INC424 - Data from multiple research programs will be presented, including two pivotal Phase III studies evaluating INC424 benefit versus placebo (COMFORT-I [COntrolled MyeloFibrosis study with ORal JAK inhibitor Therapy]) (ASH abstract #278; December 12, 7:00 - 8:30AM) and versus best available therapy (COMFORT-II) (ASH abstract #795; December 12, 4:30 - 6:00PM). These data will assess measures of spleen reduction, symptom improvement, health-related quality of life and overall survival.
Everolimus - Results from a Phase II study evaluating everolimus as a monotherapy in relapsed/refractory Hodgkin lymphoma (ASH abstract #2717; December 11, 6:00 - 8:00PM) and data from a Phase I trial of everolimus in combination with rituximab or in combination with BTZ and rituximab in relapsed/refractory Waldenström's macroglobulinemia (ASH abstract #2705; December 11, 6:00 - 8:00PM).
LBH589 - Results from PANORAMA-2 (PANobinostat ORAl in Multiple myelomA), a Phase II study of LBH589 in combination with BTZ and dexamethasone in patients with relapsed and BTZ-refractory multiple myeloma (ASH abstract #814; December 12, 4:30 - 6:00PM). Data from two trials in myelofibrosis: final results from a Phase I trial of prolonged low dose therapy with LBH589 in myelofibrosis patients (ASH abstract #794; December 12, 4:30 - 6:00PM) and a preclinical study of LBH589 in combination with INC424 in JAK2V617F-driven disease (ASH abstract #798; December 12, 4:30 - 6:00PM).
About everolimus tablets
Everolimus is approved as Afinitor® (everolimus) tablets in more than 80 countries including the United States and throughout the European Union in the oncology settings of advanced renal cell carcinoma (RCC) following progression on or after vascular endothelial growth factor (VEGF)-targeted therapy and in the US and EU for locally advanced, metastatic or unresectable progressive neuroendocrine tumors of pancreatic origin (pNET).
Everolimus is also available from Novartis for use in non-oncology patient populations under the brand names Afinitor® or Votubia®, Certican® and Zortress® and is exclusively licensed to Abbott and sublicensed to Boston Scientific for use in drug-eluting stents.
Indications vary by country and not all indications are available in every country. Access to everolimus outside of the approved indications is carefully controlled and monitored in clinical trials designed to better understand the potential benefits and risks of the compound. As an investigational compound, the safety and efficacy profile of everolimus has not yet been established outside the approved indications. Because of the uncertainty of clinical trials, there is no guarantee that everolimus will become commercially available for additional indications anywhere else in the world.
Everolimus tablets Important Safety Information
Everolimus can cause serious side effects including lung or breathing problems, infections, and renal failure which can lead to death. Mouth ulcers and mouth sores are common side effects. Everolimus can affect blood cell counts, kidney and liver function, and blood sugar and cholesterol levels. Everolimus may cause fetal harm in pregnant women. Women taking everolimus should not breast feed.
The most common adverse drug reactions (incidence >=15%) are mouth ulcers, diarrhea, feeling weak or tired, skin problems (such as rash or acne), infections, nausea, swelling of extremities or other parts of the body, loss of appetite, headache, inflammation of lung tissue, abnormal taste, nose bleeds, inflammation of the lining of the digestive system, weight decreased and vomiting. The most common Grade 3-4 adverse drug reactions (incidence >=2%) are mouth ulcers, feeling tired, low white blood cells (a type of blood cell that fights infection), diarrhea, infections, inflammation of lung tissue, and diabetes. Cases of hepatitis B reactivation and blood clot in the lung and leg have been reported.
About Zometa (zoledronic acid)
Zometa® (zoledronic acid) Injection is indicated for the prevention of skeletal-related events (SREs; pathological fractures, spinal compression, radiation or surgery to bone, or tumor-induced hypercalcemia) in patients with multiple myeloma and advanced malignancies involving bone. The recommended dose is a 4 mg, 15-minute infusion every 3-4 weeks.
Zometa Important Safety Information
Zometa has been associated with reports of renal insufficiency. Adequately rehydrate patients and assess serum creatinine prior to each dose. Single doses of Zometa should not exceed 4 mg and the duration of infusion should be no less than 15 minutes in 100 ml of dilutent. The risk of renal adverse events may be greater in patients with renal insufficiency and dose adjustments are required. Not recommended in patients with severe renal impairment. Monitor serum levels of calcium, phosphate and magnesium and treat as necessary. Severe and occasionally incapacitating bone, joint, and/or muscle pain has been reported. Monitor for thigh, hip or groin pain, evaluate for femur fractures as necessary and discontinue treatment if required. Use caution in aspirin-sensitive patients, and when using aminoglycosides, loop diuretics and other potentially nephrotoxic drugs. Use caution in multiple myeloma patients using thalidomide. Patients being treated with Zometa should not be treated with Aclasta(3) concomitantly. Zometa should not be used in patients who are pregnant, or plan to become pregnant, or who are breast-feeding. Contraindicated in patients with hypersensitivity to zoledronic acid, other bisphosphonates, or any of the excipients in Zometa.
In clinical trials, the most commonly reported adverse events included flu-like syndrome (fever, arthralgias, myalgias, skeletal pain), fatigue, gastrointestinal reactions, anemia, weakness, cough, dyspnea and edema. Osteonecrosis of the Jaw (ONJ): ONJ has been reported in patients with cancer receiving treatment including bisphosphonates, chemotherapy, and/or corticosteroids. The majority of reported cases have been associated with dental procedures such as tooth extraction. A dental examination with appropriate preventive dentistry should be considered prior to treatment with bisphosphonates in patients with concomitant risk factors. While on treatment, these patients should avoid invasive dental procedures if possible. No data are available to suggest whether discontinuation of bisphosphonate therapy reduces the risk of ONJ in patients requiring dental procedures. A causal relationship between bisphosphonate use and ONJ has not been established.
Please see full Prescribing Information. Approved indications vary by country.
About BKM120 and LBH589 (panobinostat)
Because these are investigational compounds, the safety and efficacy profile of BKM120 and LBH589 have not yet been established. Access to these investigational compounds is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the compound. Because of uncertainty of clinical trials, there is no guarantee that BKM120 and LBH589 will ever be commercially available anywhere in the world.
About Tasigna (nilotinib)
Tasigna® (nilotinib) is approved in more than 90 countries for the treatment of chronic phase and accelerated phase Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML) in adult patients resistant or intolerant to at least one prior therapy, including Glivec, and for the treatment of adult patients with newly diagnosed Ph+ CML in chronic phase. Take twice daily 12 hours apart. Do not take with food. No food to be consumed for 2 hours before or one hour after dosing. Avoid grapefruit juice and CYP3A4 inhibitors.
Tasigna Important Safety Information
Use with caution in patients with uncontrolled or significant cardiac disease and in patients who have or may develop prolongation of QTc. Low levels of potassium or magnesium must be corrected prior to Tasigna administration. Monitor closely for an effect on the QTc interval. Baseline ECG is recommended prior to initiating therapy and as clinically indicated. Uncommon cases (0.1 to 1%) of sudden death have been reported in clinical studies in patients with significant risk factors.
Use with caution in patients with liver impairment, with a history of pancreatitis and with total gastrectomy. Patients with rare hereditary problems of galactose intolerance, severe lactase deficiency or glucose-galactose malabsorption should not use Tasigna. Tasigna may cause fetal harm in pregnant women. Women taking Tasigna should not breastfeed.
The most frequent Grade 3 or 4 adverse events are hematological (neutropenia and thrombocytopenia) which are generally reversible and usually managed by withholding Tasigna temporarily or dose reduction. Monitor blood counts regularly. Pancreatitis has been reported. The most frequent non-hematologic adverse events were rash, pruritus, nausea, fatigue, headache, alopecia, myalgia, constipation and diarrhea. Most of these adverse events were mild to moderate in severity.
Please see full Prescribing Information.
About Glivec (imatinib)
Glivec® (imatinib) is approved in more than 110 countries for the treatment of all phases of Ph+ CML, for the treatment of adult patients with KIT (CD117)-positive gastrointestinal stromal tumors (GIST), which cannot be surgically removed and/or have metastasized and for the treatment of adult patients following complete surgical removal of KIT+ GIST. Take with food and a large glass of water.
Glivec Important Safety Information
Glivec can cause fetal harm in pregnant woman. Glivec has been associated with severe edema (swelling) and serious fluid retention. Cytopenias (anemia, neutropenia, thrombocytopenia) are common, generally reversible and usually managed by withholding Glivec or dose reduction. Monitor blood counts regularly. Severe congestive heart failure and left ventricle dysfunction, severe liver problems including cases of fatal liver failure and severe liver injury requiring liver transplants have been reported. Use caution in patients with cardiac dysfunction and hepatic dysfunction. Monitor carefully.
Bleeding may occur. Severe gastrointestinal (GI) bleeding has been reported in patients with KIT+ GIST. Skin reactions, hypothyroidism in patients taking levothyroxine replacement, GI perforation, in some cases fatal and tumor lysis syndrome, which can be life threatening, have also been reported with Glivec. Correct dehydration and high uric acid levels prior to treatment. Long-term use may result in potential liver, kidney, and/or heart toxicities; immune system suppression may also result from long-term use. In patients with hypereosinophilic syndrome and heart involvement, cases of heart disease have been associated with the initiation of Glivec therapy. Growth retardation has been reported in children taking Glivec. The long-term effects of extended treatment with Glivec on growth in children are unknown.
The most common side effects include fluid retention, muscle cramps or pain and bone pain, abdominal pain, loss of appetite, vomiting, diarrhea, decreased hemoglobin, abnormal bleeding, nausea, fatigue and rash.
Please see full Prescribing Information.
About Exjade (deferasirox)
Exjade® (deferasirox) is indicated for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in adult and pediatric patients (aged 2 years and over). It is approved in over 100 countries including the U.S., Switzerland, Japan, and the countries comprising the European Union. The approved indication may vary depending upon the individual country.
Exjade Important Safety Information
Exjade is contraindicated in patients with an estimated creatinine clearance <60 mL/min, with hypersensitivity to the active substance or any of the excipients, or in combination with other iron chelator therapies. Exjade is not recommended in patients with severe hepatic impairment.
There have been postmarketing reports of acute renal failure, hepatic failure, and cytopenias. Renal failure requiring temporary or permanent dialysis, renal tubulopathy, and interstitial nephritis have been reported. Upper gastrointestinal ulceration and hemorrhage, sometimes fatal, have been reported. Caution should be used in elderly patients due to a higher frequency of adverse reactions. Exjade is not recommended in patients with a short life expectancy (e.g., high-risk myelodysplastic syndromes), especially when co-morbidities could increase the risk of adverse events.
Skin rashes, serious hypersensitivity reactions, decreased hearing, and lens opacities have been reported. The most common adverse reactions are nausea, vomiting, diarrhea, abdominal pain, rash, non-progressive increases in serum creatinine, increased transaminases, abdominal distension, constipation, dyspepsia, proteinuria, and headache.
Please visit www.exjade.com for more information.
About INC424 (ruxolitinib)
INC424 (ruxolitinib) is an oral inhibitor, of the JAK1 and JAK2 tyrosine kinases. INC424 is being investigated in primary myelofibrosis as well as post-polycythemia vera myelofibrosis (PPV-MF) and post-essential thrombocythemia myelofibrosis (PET-MF). INC424 is also being investigated in clinical trials for the treatment of polycythemia vera (PV).
Novartis licensed INC424 from Incyte for development and potential commercialization outside the US. Incyte has retained rights for the development and potential commercialization of INC424 in the US. Both the US Food and Drug Administration and the European Medicines Agency have granted INC424 orphan drug status for myelofibrosis.
(1) Novartis and Incyte Corporation have a worldwide collaboration and licensing agreement for INC424.
(2) Known as Gleevec® (imatinib mesylate) tablets in the US, Canada and Israel.
(3) Known as Reclast in the US.
The foregoing release contains forward-looking statements that can be identified by terminology such as "will," "goal," "potential," or similar expressions, or by express or implied discussions regarding potential new indications or labeling, or potential marketing approvals for Novartis Oncology products, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that any additional indications or labeling will be approved for any existing Novartis Oncology products, or that any new Novartis Oncology products will be approved for sale in any market, or at any particular time. Nor can there be any guarantee that any such products will achieve any particular levels of revenue in the future. In particular, management's expectations could be affected by, among other things, unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; unexpected manufacturing issues; the impact that the foregoing factors could have on the values attributed to the Novartis Group's assets and liabilities as recorded in the Group's consolidated balance sheet, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
Business and Industry | Breaking World Pharma News
Molecule-making machine simplifies complex chemistry
Friday, 13 March 2015
A new molecule-making machine could do for chemistry what 3-D printing did for engineering: Make it fast, flexible and accessible to anyone. Chemists at the University of Illinois, led by chemistry professor and medical doctor Martin D. Burke, built the machine to assemble complex small molecules at the click of a mouse, like a 3-D printer at the molecular level.
A new molecule-making machine could do for chemistry what 3-D printing did for engineering: Make it fast, flexible and accessible to anyone. Chemists at the University of Illinois, led by chemistry professor and medical doctor Martin D. Burke, built the machine to assemble complex small molecules at the click of a mouse, like a 3-D printer at the molecular level. The automated process has the potential to greatly speed up and enable new drug development and other technologies that rely on small molecules.
"We wanted to take a very complex process, chemical synthesis, and make it simple," said Burke, a Howard Hughes Medical Institute Early Career Scientist. "Simplicity enables automation, which, in turn, can broadly enable discovery and bring the substantial power of making molecules to nonspecialists."
The researchers described the technology in a paper featured on the cover of the March 13 issue of Science.
"Small molecules" are a specific class of complex, compact chemical structures found throughout nature. They are very important in medicine - most medications available now are small molecules - as well as in biology as probes to uncover the inner workings of cells and tissues. Small molecules also are key elements in technologies like solar cells and LEDs.
However, small molecules are notoriously difficult to make in a lab. Traditionally, a highly trained chemist spends years trying to figure out how to make each one before its function can even be explored, a slowdown that hinders development of small-molecule-based medications and technologies.
"Up to now, the bottleneck has been synthesis," Burke said. "There are many areas where progress is being slowed, and many molecules that pharmaceutical companies aren't even working on, because the barrier to synthesis is so high."
The main question that Burke's group seeks to answer: How do you take something very complex and make it as simple as possible?
The group's strategy has been to break down the complex molecules into smaller building blocks that can be easily assembled. The chemical building blocks all have the same connector piece and can be stitched together with one simple reaction, the way that a child's interconnecting plastic blocks can have different shapes but all snap together. Many of the building blocks Burke's lab has developed are available commercially.
To automate the building-block assembly, Burke's group devised a simple catch-and-release method that adds one building block at a time, rinsing the excess away before adding the next one. They demonstrated that their machine could build 14 different classes of small molecules, including ones with difficult-to-manufacture ring structures, all using the same automated building-block assembly.
"Dr. Burke's research has yielded a significant advance that helps make complex small molecule synthesis more efficient, flexible and accessible," said Miles Fabian of the National Institutes of Health's National Institute of General Medical Sciences, which partially funded the research. "It is exciting to think about the impact that continued advances in these directions will have on synthetic chemistry and life science research."
The automated synthesis technology has been licensed to REVOLUTION Medicines, Inc., a company that Burke co-founded that focuses on creating new medicines based on small molecules found in nature. The company initially is focusing on anti-fungal medications, an area where Burke's research has already made strides.
"It is expected that the technology will similarly create new opportunities in other therapeutic areas as well, as the industrialization of the technology will help refine and broaden its scope and scalability," Burke said.
"Perhaps most exciting, this work has opened up an actionable roadmap to a general and automated way to make most small molecules. If that goal can be realized, it will help shift the bottleneck from synthesis to function and bring the power of making small molecules to nonspecialists."
Astellas and Ambrx initiate collaboration for discovery and development of antibody drug conjugates for oncology
Monday, 08 April 2013
Astellas Pharma Inc. (Tokyo: 4503, "Astellas") entered into a collaboration with Ambrx Inc. ("Ambrx") for the discovery and development of novel antibody drug conjugates ("ADCs"). ADCs allow for the targeted delivery of drugs to the target tissue. Ambrx creates optimized ADCs using its site-specific conjugation technology along with proprietary linkers and payloads. In the preclinical setting, Ambrx ADCs have demonstrated high potency and a wider therapeutic index than ADCs created using conventional non-specific conjugation.
Ambrx will receive an upfront payment of $15 million from Astellas, as well as up to $285 million in potential near and long-term research, development, regulatory and sales-based milestones for an undisclosed number of targets for ADCs in oncology. A portion of these milestones, as well as royalties on any net sales, will be contingent on eventual successful commercialization of products developed as a result of this partnership. Astellas will receive worldwide rights to develop and commercialize ADCs for oncology. Additional terms of the collaboration are not disclosed at this time.
"Agensys, Inc., an affiliate of Astellas which specializes in therapeutic antibody research and development in cancer, has significant experience with ADCs as oncology therapeutics and is looking to further expand its capabilities in this area. Ambrx offers a novel approach to allow creation of site-specific and highly stable conjugations that have the potential to further optimize drug delivery to tumor cells." said David Stover, Ph.D., Senior Vice President and Agensys Site Head.
Added Lawson Macartney, chief executive officer of Ambrx, "We recognize Astellas as a leader in the development of innovative therapeutics for oncology and are proud to initiate this collaboration. We look forward to developing these therapeutics while also advancing our broad pipeline of partnered and wholly owned therapeutic candidates with best-in-class conjugation."
Ambrx Inc. is a clinical stage biopharmaceutical company using an expanded genetic code to create best-in-class bio therapeutics, including antibody drug conjugates and proteins with improved pharmacologic properties. The company is developing ARX201, a long-acting growth hormone that has successfully completed Phase 2b clinical trials. Ambrx has collaborations to discover and develop products incorporating Ambrx technology with Bristol-Myers Squibb, Eli Lilly and Company and several undisclosed companies. Ambrx is advancing a robust portfolio of product candidates that are optimized for efficacy, safety and ease of use in multiple therapeutic areas.
Astellas Pharma Inc., located in Tokyo, Japan, is a pharmaceutical company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceuticals. Astellas has approximately 17,000 employees worldwide. The organization is committed to becoming a global category leader in Urology, Immunology (including Transplantation) and Infectious Diseases, Oncology, Neuroscience and DM Complications and Kidney Diseases.
Conferences and Events
Doctors 2.0 & You to address the "explosion of digital health tools" with new subjects, speakers, partners
Tuesday, 18 March 2014
5 - 6 June 2014, Paris, France.
Doctors 2.0 & You, partner of Stanford Medicine X and known for its 360° inclusive perspective on the patient, the physician, and digital tools, today announced 4th edition highlights. Remarking that "healthcare is now a recognized part of the explosion in social media, apps, quantified self and wearables," founder Denise Silber, confirms that "the June, 2014 program will provide an intensive look at the many moving parts of digital health and their results to date. How do patients and physicians benefit?"
Doctors 2.0 & You will bring attendees the latest insights and research on the ePatient and the digital doctor, individually and in communities, social media campaigns, eReputation, mobile apps, Serious Games and simulation, Quantified self and wearables including Google Glass and sensors. These tools will be envisaged from the standpoint of patients and physicians and other key stakeholders: pharma and device industries and suppliers, technology companies, payers, government. Participants originate from around the world and provide both a global and national perspective.
"So many people keep talking about digital, 2.0, social media... applied to the healthcare world. Doctors 2.0 & You helps make it happen, by spotting trends and talent and bringing la crème de la crème together. Doctors 2.0 & You is a best practice in Europe,” said Bernard Peyrical, Sanofi.
Some highlights from the 20 hours of activities including 22 parallel sessions and plenary, and 6 hours of Networking
- Connected Objects, Quantified Self: Special activities around connected objects and quantified self will be developed for attendees thanks to the new Platinum Partnership with iHealthLabs Europe, founded by Uwe Diegel. Other quantified self players, ePatient and physician users, and pharma (Novartis) will provide their testimonials.
- Google Glass: Four international pioneer Google Glass explorers will present exciting perspectives on health and medicine. Diversifying digital pharma
- Pharma partners from hospitals and patient and physician communities will discuss relationships and results.
- Dr Berci Mesko, founder of Webicina and medical futurist, will provide a limited seat Master Class on quality of web resources.
- Award winning mobile apps and social media campaigns from Europe and the US will explain how they did it.
- Three authors from the US, Hungary, and France will sign their books at the June 5th networking reception.
- Stanford Medicine X and Doctors 2.0 & You will sponsor a medical student leader participant.
- Announcement of the Doctors 2.0 & You Start Up Finalist who will fly to Palo Alto in September to present
- Returning partners Novartis, Sanofi, LexisNexis and DocCheck will provide exciting activities in the exhibit hall.
- Doctors 2.0 & You welcomes new supporting partners Ben's Friends and SOPE (Society of Physician Entrepreneurs).
Research and Development | Clinical Trials
Affinium Pharmaceuticals announces completion of recruitment for Phase 2 clinical trial of AFN-1252
Thursday, 09 August 2012
Affinium Pharmaceuticals announced that full recruitment has been achieved in its multi-center Phase 2 clinical trial evaluating oral AFN-1252 in acute bacterial skin & skin structure infections ("ABSSSI"). This Phase 2 trial is the first human efficacy study conducted with a new class of an tibiotics designed to specifically inhibit staphylococcal fatty acid biosynthesis via a new drug target, the fatty acid synthase II (FASII) system. Results expected in late 2012 will represent a significant proof-of-concept milestone for this unique, Staphylococcus-specific antibiotic, AFN-1252.
The objectives of the study are to confirm efficac y, safety and tolerability of 200 mg of oral AFN-1252 dosed twice daily for 5-14 days in patients with serious, suspected staphylococcal skin infections recruited in the outpatient or emergency room settings. The trial evaluates both the traditional endpoints at end of treatment a nd early endpoints currently recommended by the FDA.
"We are delighted with the ease and rate of patient recruitment in our study," said Dr. Barry Hafkin, Chief Medical Officer at Affinium. Dr. Hafkin noted that investigators had the option of adding a second antibiotic to cover any other pot ential pathogen or admitting the patient into hospital; yet, the majority of the patients we re treated with AFN-1252 as monotherapy in the outpatient setting. "We believe this study demonstrates investigators' confidence in identifying staphylococcal skin infection and using AFN-1252 in the treatm ent of serious staphylococcal skin infections. The exquisite potency of AFN-1252 agains t all strains of Staphylococcus including MRSA and VISA through a unique new m echanism of action, and the excellent safety profile demonstrated in multiple oral Phase 1 studies were attributes that excited our clinical investigators," Dr. Hafkin noted.
According to Leisa Dennehy, Commercial & Corporate Development Advisor at Affinium, "Practicing physicians and key opi nion leaders consistently remark that a specific-spectrum agent has unique and important benefits that no other antibiotic has ever been able to offer." Robert Daum, M.D., Director of the Univers ity of Chicago MRSA Research Center and Professor of Pediatrics, Microbiology and Molecular Medicine at the University of Chicago, Illinois, commented: "A staphylococcal specific antibiotic should have no off-target effect on gut flora and put no resistance selection pressure on other bacterial spec ies, which may greatly reduce the probability of antibiotic associated adverse events such as C. difficile disease, diarrhea or candidiasis. The incredible potency against all strains of Staphylococcus combined with an excellent safety profile demonstrated in multip le oral Phase 1 makes AFN-1252 an exciting new product for treatment of staphylococcal infections."
About Staphylococcal Infections
Each year in the US, more people die from staphylococcal infections that from HIV. Skin infections alone account for more ten million patient visits in the US. Staphylococcus is the mostly commonly identified bacterium in skin infections and is a poten tial pathogen in almost every infection type, making staphylococci the most common bacterial pathogen in a large and diverse patient population.
AFN-1252 is the lead clinical-stage agent in a new class of antibiotics base d on FabI inhibition. AFN-1252 is a well tolerated and highly potent agent that is ac tive against all strains of Staphylococcus, including all known resistant strains such as methicillin-resistant S.aureus (MRSA) and vancomycin-intermediate S.aureus (VISA). Because AFN-1252 is specifically designed to be active against only Staphylococcus sp., it may provide the unique safety benefit of no off-target side effects, such as antibiotic-induced diarrhea or resistance pressure on other bacteria. AFN-1252 has potential to be used eith er as monotherapy for confirmed staphylococcal infections or in combination when Staphylococcus is suspected in a variety of infections. The oral formulation is finishing Ph ase 2a, and the IV formulation is completing pre-clinical studies in 2012.
About Affinium Pharmaceuticals
Affinium Pharmaceuticals is a specialty pharmaceutical company focused on the development of novel anti-infective medicines. The Affinium fatty acid synthesis ("FASII") antibacterial program constitutes a new antibiotic franchise with the potential for multiple patented products targeting the FASII pathway in various bacteria.
Drug not Shown to be Safe and Effective in Breast Cancer Patients
Tuesday, 22 November 2011
The U.S. Food and Drug Administration
FDA Commissioner Margaret A. Hamburg, M.D., said she is revoking the agency's approval of the breast cancer indication for Avastin (bevacizumab) after concluding that the drug has not been shown to be safe and effective for that use.
Avastin will still remain on the market as an approved treatment for certain types of colon, lung, kidney and brain cancer (glioblastoma multiforme).
"This was a difficult decision. FDA recognizes how hard it is for patients and their families to cope with metastatic breast cancer and how great a need there is for more effective treatments. But patients must have confidence that the drugs they take are both safe and effective for their intended use," Dr. Hamburg said. "After reviewing the available studies it is clear that women who take Avastin for metastatic breast cancer risk potentially life-threatening side effects without proof that the use of Avastin will provide a benefit, in terms of delay in tumor growth, that would justify those risks. Nor is there evidence that use of Avastin will either help them live longer or improve their quality of life."
Avastin's risks include severe high blood pressure; bleeding and hemorrhaging; heart attack or heart failure; and the development of perforations in different parts of the body such as the nose, stomach, and intestines.
The decision, outlined in Dr Hamburg's 69-page opinion, involves Avastin used in combination with the cancer drug paclitaxel for those patients who have not been treated with chemotherapy for their form of metastatic breast cancer known as HER2 negative. This indication must now be removed from Avastin's product labeling.
Dr. Hamburg's decision is based on an extensive record, which includes thousands of pages submitted to a public docket, data from several clinical trials and the record from a two-day hearing held in June, 2011.
Avastin was approved for metastatic breast cancer in February 2008 under the FDA's accelerated approval program, which allows a drug to be approved based on data that are not sufficiently complete to permit full approval. The accelerated approval program provides earlier patient access to promising new drugs to treat serious or life-threatening conditions while confirmatory clinical trials are conducted. If the clinical trials do not justify the continued approval of the drug or a specific drug indication, the agency may revoke its approval. In this case, the accelerated approval was based on promising results from one study that suggested that the drug could provide a meaningful increase in the amount of time from when treatment is started until the tumor grows or the death of the patient.
After the accelerated approval of Avastin for breast cancer, the drug's sponsor, Genentech, completed two additional clinical trials and submitted the data from those studies to the FDA. These data showed only a small effect on tumor growth without evidence that patients lived any longer or had a better quality of life compared to taking standard chemotherapy alone - not enough to outweigh the risk of taking the drug.
FDA's Center for Drug Evaluation and Research, which is responsible for the approval of this drug, ultimately concluded that the results of these additional studies did not justify continued approval and notified Genentech it was proposing to withdraw approval of the indication.
Genentech did not agree with the Center's evaluation of the data and, following the procedures set out in FDA regulations, requested a hearing on the Center's withdrawal proposal, with a decision to be made by the Commissioner. That two-day hearing, which took place June 28-29, 2011, included recommendations from the FDA's Oncologic Drugs Advisory Committee (ODAC), voting 6-0 in favor of withdrawing approval of Avastin's breast cancer indication. After the hearing, the public docket remained open until Aug. 4, 2011. (In an earlier meeting of the ODAC, that committee had voted 12-1 in favor of the removal of the breast cancer indication from the Avastin label).
"FDA is committed to working with sponsors to bring promising cancer drugs to market as quickly as possible using tools like accelerated approval," Dr. Hamburg said. "I encourage Genentech to consider additional studies to identify if there are select subgroups of women suffering from breast cancer who might benefit from this drug."