PTC Therapeutics, Inc. (NASDAQ: PTCT) recently announced the withdrawal of the new drug application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following feedback from the U.S. Food and Drug Administration (FDA). The company's Chief Executive Officer, Matthew B. Klein, stated that the FDA shared that based on its review, the data in the NDA submission are unlikely to meet the agency’s threshold of substantial evidence of effectiveness to support approval of Translarna.
Translarna™ (ataluren) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. Duchenne muscular dystrophy (Duchenne) is a rare and fatal genetic disorder primarily affecting males, which results in progressive muscle weakness from early childhood and leads to premature death in the mid-20s due to heart and respiratory failure.
PTC Therapeutics, Inc. is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of clinically differentiated medicines for children and adults living with rare disorders. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders.
There was no specific financial or metric information provided in the press release to compare with previous periods. Today the company's shares have moved -4.18% to a price of $72.48. Check out the company's full 8-K submission here.
