PTC THERAPEUTICS, INC. (NASDAQ:PTCT) Files An 8-K Other EventsItem 8.01. Other Events.
On August 27, 2018, Akcea Therapeutics, Inc. (“Akcea”), an affiliate of Ionis Pharmaceuticals, Inc. (“Ionis”), and Ionis issued a press release stating that Akcea and Ionis received a Complete Response Letter from the Division of Metabolism and Endocrinology Products of the U.S. Food and Drug Administration regarding the New Drug Application for WAYLIVRA™ (volanesorsen) for the treatment of people with familial chylomicronemia syndrome (“FCS”). WAYLIVRA is also under regulatory review in the E.U. and Canada for the treatment of people with FCS.
As previously disclosed, WAYLIVRA, an antisense drug candidate in development for two rare metabolic disorders, FCS and familial partial lipodystrophy (FPL), is subject to a collaboration and license agreement by and between Akcea and PTC Therapeutics International Limited (“PTC”), a subsidiary of PTC Therapeutics, Inc., to which, among other things, Akcea granted PTC an exclusive right and license to develop, manufacture and commercialize WAYLIVRA in countries in Latin America and the Caribbean.
About PTC THERAPEUTICS, INC. (NASDAQ:PTCT)
PTC Therapeutics, Inc. is a biopharmaceutical company focused on the discovery, development and commercialization of orally administered, small molecule therapeutics that focus on post-transcriptional control processes. The Company’s lead product, Translarna (ataluren), is used for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients with age of over five years and older. Its programs include Translarna for nonsense mutation cystic fibrosis (nmCF), Translarna for mucopolysaccharidosis type I caused by nonsense mutation (nmMPS I), Translarna for nonsense mutation aniridia, Translarna for nonsense mutation Dravet syndrome/CDKL5, Spinal muscular atrophy (SMA) in collaboration with Spinal Muscular Atrophy Foundation (SMA Foundation) and F. Hoffman-La Roche Ltd and Hoffman- La Roche Inc. (collectively Roche), and Cancer stem cell program (PTC596). It has two compounds in clinical development within the SMA program: RG7800 and RG7916.
