PTC THERAPEUTICS, INC. (NASDAQ:PTCT) Files An 8-K Other EventsItem 8.01.Other Events
On September 28, 2017, the U.S. Food and Drug Administration’s (FDA) Peripheral and Central Nervous Systems Drugs Advisory Committee (PCNS) met to discuss ataluren (Translarna™) for the treatment of nonsense mutation dystrophinopathy. The single question to the advisory committee was “The best interpretation of the information presented today regarding the use of ataluren for the treatment of dystrophinopathies resulting from nonsense mutations in the dystrophin gene is that:…".
The vote was cast as follows:
No votes for “the data suggest that ataluren is not effective.”
10 votes for “although it is possible that ataluren may be effective, the data are inconclusive, and more work would be needed to establish whether ataluren is effective.”
1 vote for “the data are sufficient to conclude that ataluren is effective.”
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.
