According to a press release put out by PTC Therapeutics, Inc., the Office of Drug Evaluation of the Food and Drug Administration on Wednesday, Oct. 25th, 2017, sent a complete response letter (CRL) to that biopharmaceutical company, denying a New Drug Application (NDA) for its investigational study drug ataluren used to treat Duchenne muscular dystrophy (DMD).
ANOTHER CONTROLLED STUDY REQUIRED
Duchenne muscular dystrophy is caused by a lack of dystrophin and ataluren was a promising investigational study drug in the United States that was supposed to help treat young men with the DMD. Ataluren is marketed under the trade name Translarna in Europe.
Basically, the FDA said the drug doesn’t work. The letter read in part that, “evidence of effectiveness from an additional adequate and well-controlled clinical trial(s) will be necessary at a minimum to provide substantial evidence of effectiveness.”
PARENT PROJECT MUSCULAR DYSTROPHY RESPONDS
Parent Project Muscular Dystrophy (PPMD) is a U.S-based nonprofit organization founded in 1994 and is devoted to curing Duchenne muscular dystrophy. PPMD’s community disagrees with the outcome.
PPMD acknowledged the uphill climb for ataluren, noting that the NDA was filed “over protest” by the FDA. The FDA did not approve of the data package submitted by PTC and sought its rejection on that point before submission. PTC submitted the NDA anyway.
PPMD responded to the FDA’s findings in a post on their Facebook Page:
“This decision is disappointing and not the outcome that our community hoped it would be. However, today’s decision is not the end of the ataluren story.”
— STATEMENT FROM PARENT PROJECT MUSCULAR DYSTROPHY
PPMD pledged to support PTC Therapeutics while the company gathers the necessary data for their filing of formal dispute resolution request. PTC Therapeutics has indicated their desire to file the dispute next week.
PPMD has supported the work of PTC Therapeutics in developing ataluren, noting that many of the young men in the trial have been in the trial for 10 years, over half of their lives. PPMD is working to secure continued access to ataluren while figuring out what role their support may take during the process of disputing the committee’s findings.
Stay tuned for more developments in future posts.
