Gazyva regulatory update
Canadian Minister of Health Rona Ambrose announced the launch of a pilot project that solicits input from patients with rare diseases to guide future reviews of orphan drugs. Canada is in the process of implementing orphan regulations since a designation was authorized in 2012. Patients will be asked how their disease affects their daily lives, what treatments are currently available, what therapeutic benefits are most important to them, and what their risk tolerance is for new treatments. The pilot project will simulate how input from patients will be gathered and incorporated into the drug review process once the Orphan Drug Framework is in effect. Obinutuzumab for treating chronic lymphocytic leukemia (CLL) from Roche and Ravicti glycerol phenylbutyrate to treat urea cycle disorders (UCD) from Hyperion Therapeutics Inc. (NASDAQ:HPTX, South San Francisco, Calif.) will be included in the pilot project.
Ravicti, a pre-prodrug of phenylacetic acid, is under review in Europe and Canada to treat UCD. Ravicti is approved in the U.S. for the management of chronic UCD in patients ages >=2 years. The product has Orphan Drug designation in the U.S. and Europe for maintenance treatment of patients with UCD and in the U.S. for the intermittent or chronic treatment of patients with cirrhosis and any grade of hepatic encephalopathy (HE). Hyperion has worldwide rights to the product from Ucyclyd Pharma Inc., a subsidiary of Medicis Pharmaceutical Corp., which Valeant Pharmaceuticals International Inc. (TSX:VRX; NYSE:VRX, Montreal, Quebec) acquired in 2012 (see BioCentury, April 2, 2012 & Dec. 17, 2012). ...