Monday, May 2, 2016
Sanofi U.S. has announced collaborations with the Duke Clinical Research Institute (DCRI) and, separately, with the Center for Assessment Technology and Continuous Health (CATCH) at Massachusetts General Hospital. The collaborations are designed to create new tools that will help predict how people living with type 2 diabetes adhere to their medication.
Abbott has signed a definitive agreement to acquire St. Jude Medical, creating a medical device provider with a higher position in high-growth cardiovascular markets, including atrial fibrillation, structural heart and heart failure, as well as a position in the high-growth neuromodulation market.
Inovio Pharmaceuticals has announced that its immunotherapy for hepatitis C (INO-8000) will be evaluated in a phase I trial in chronically infected patients who are not receiving other hepatitis C virus (HCV) treatments. The study will enroll patients who are in the early stages of chronic HCV infection to determine the therapy’s ability to decrease and potentially eliminate HCV viral load, measure HCV specific immune responses and durability of these immune responses, and evaluate safety and tolerability. In this dose-escalation study INO-8000 will be combined with increasing doses of DNA-based IL-12 (INO-9012), an immune activator, which in previous studies has been shown to increase the therapeutic immune response to DNA immunotherapies.
The FDA has granted three Breakthrough Therapy designations for Novartis’ Ilaris (canakinumab) to treat three rare types of Periodic Fever Syndromes, also known as Hereditary Periodic Fevers. Novartis will work closely with the FDA to expedite the regulatory review of Ilaris for these conditions.
Periodic Fever Syndromes are a group of autoinflammatory diseases that cause disabling and recurrent fevers, which may be accompanied by joint pain and swelling, muscle pain and skin rashes, with complications that can be life-threatening. Most patients present with symptoms in infancy or childhood.
The three conditions for which Ilaris is being reviewed are Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), and in Familial Mediterranean Fever (FMF) patients who are not adequately controlled with colchicine.
“This is an important day for patients, including many children, who are affected by these serious and debilitating syndromes that have no or limited treatment options,” said David Epstein, division head, Novartis Pharmaceuticals. “Ilaris is a promising medicine under review for these conditions, marking our commitment to making a significant difference to the lives of people with rare diseases.”
The FDA considers a treatment a Breakthrough Therapy if it is intended to treat a serious or life-threatening condition and preliminary evidence indicates it may be better than existing treatments. If approved, Ilaris will likely be the first medicine to gain approval from drug regulators for the treatment of TRAPS and HIDS/MKD, and it will be an alternative to the only FDA-approved treatment for FMF, colchicine.
The Breakthrough Therapy Designations were granted based on the pivotal phase III trial (Canakinumab Pivotal Umbrella Study in Three Hereditary Periodic Fevers). Based on this study, Novartis submitted three supplemental Biologic License Applications in the U.S. to register Ilaris for use in these indications.
Ilaris was approved by the FDA in 2009 to treat two subtypes of a rare autoinflammatory disease called Cryopyrin-Associated Periodic Syndromes (CAPS): Muckle-Wells syndrome (MWS) and Familial Cold Autoinflammatory Syndrome (FCAS), in patients aged four and older. In 2013, the FDA approved Ilaris for a rare, autoinflammatory form of juvenile idiopathic arthritis called Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged two and older.
The FDA has accepted for review the resubmission of Lundbeck’s New Drug Application (NDA) for intravenous Carnexiv, an intravenous formulation of the anti-epileptic drug (AED) carbamazepine. An action letter is anticipated before the end of 2016. Lundbeck’s resubmission was in reply to the Complete Response Letter from the FDA issued in 2014 requesting additional data associated with the Chemistry, Manufacturing and Controls (CMC) of the product. The proposed U.S. trade name, Carnexiv, is under consideration with the FDA as well.
Sanford Burnham Prebys Medical Discovery Institute (SBP), a fundamental and translational research institute, and GlaxoSmithKline (GSK), a global pharmaceutical company, have announced the creation of the SBP-GSK Center for Translational Neuroscience.
DelMar Pharmaceuticals has announced that the FDA Office of Orphan Products Development (OOPD) has granted Orphan Drug designation for its lead product candidate, VAL-083, in the treatment of ovarian cancer. The investigational drug candidate previously received an orphan designation for glioma and medulloblastoma in the U.S. and glioma in Europe.