- New Jersey-based PTC Therapeutics will lay off approximately 18% of its workforce, mainly in the U.S., the company announced on Wednesday. One month ago, the FDA issued a "refusal to file" letter in response to PTC's application for its Duchenne's muscular dystrophy (DMD) drug, Translarna.
- The FDA's letter indicated PTC's application was "not sufficiently complete" to merit a review. Translarna treats a specific mutation of DMD which prematurely stops production in the body of an essential protein.
- PTC said it would work to continue the drug's global commercialization, despite the layoffs. Translarna received conditional approval in the E.U. in 2014.
While PTC said it would work with the FDA to determine the best path forward for bringing Translarna to the U.S., PTC's immediate U.S. plans would appear to be on hold.
In a statement, PTC tied receipt of the FDA's letter to the layoffs. "This reduction is part of PTC's program intended to optimally manage operating expenses following its recent setback related to the Refuse to File letter received from the U.S. Food and Drug Administration," the company said.
The Duchenne patient community has been disappointed in 2016 as the FDA has also rejected BioMarin's dirsapersen and criticized Sarepta's eteplirsen in a preliminary staff report. There is still no approved DMD treatment in the U.S., although Translarna is approved in the E.U.