PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the New Drug Application (NDA) for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). This decision marks a significant step forward in providing a potential treatment option for boys and young men living with this genetic disorder in the United States.
Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, stated, "The NDA acceptance for review is a significant milestone that brings us one step closer to providing this important treatment to boys and young men living with nonsense mutation Duchenne muscular dystrophy in the United States. The totality of evidence clearly supports the favorable safety profile and short- and long-term benefits of Translarna for individuals with nmDMD. We look forward to working with FDA throughout the review process."
The NDA resubmission is based on findings from the global placebo-controlled trial Study 041 (N=359). The study demonstrated significant benefits in the ITT population following 72 weeks of Translarna treatment. Key study endpoints, including six-minute walk distance (6MWD) (p=0.0248), NorthStar Ambulatory Assessment (p=0.0283), 10-meter walk/run (p=0.0422), 4-stair climb (p=0.0293), and time to 10% worsening of 6MWD (p=0.0078), all showed statistically significant improvements.
Long-Term Benefits Demonstrated in STRIDE Registry
The NDA also incorporates findings from the STRIDE registry, which captured significant long-term benefits of Translarna treatment. Data from the registry indicated that Translarna treatment resulted in a 3.5-year delay in loss of ambulation (p<0.0001).
Community Support and Perspective
Pat Furlong, Founder and CEO of Parent Project Muscular Dystrophy, commented, "We thank FDA for accepting the Translarna NDA for review. Translarna uniquely addresses individuals with Duchenne muscular dystrophy secondary to nonsense mutation and could provide an important treatment option for our community. Families in the United States have been waiting a long time for a treatment that targets the underlying cause of nonsense mutation Duchenne."
