PTC Therapeutics has withdrawn its New Drug Application for ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) after the U.S. Food and Drug Administration (FDA) indicated that the submitted data are unlikely to meet the agency’s standard for substantial evidence of effectiveness, according to a press release published recently by the company.
As a result, ataluren will not be approved in the United States at this time for patients living with this specific form of DMD.
The company announced the decision following FDA feedback during its review of the resubmitted application. According to PTC Therapeutics, the agency shared that, based on its review to date, the evidence provided was unlikely to satisfy the threshold required to support approval of ataluren for nonsense mutation DMD.
“FDA shared that based on its review to date, the data in the NDA submission are unlikely to meet the Agency’s threshold of substantial evidence of effectiveness to support approval of Translarna. We have therefore made the decision to withdraw the NDA submission,” said Dr. Matthew B. Klein, chief executive officer of PTC Therapeutics. “We have worked tirelessly for over two decades to develop a safe and effective therapy for boys and young men affected by nonsense mutation DMD in the U.S. and are disappointed that FDA approval cannot be achieved.”
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Ataluren is designed as a protein restoration therapy for genetic diseases such as nonsense mutation DMD. A nonsense mutation is a change in the genetic code that prematurely stops the production of an essential protein. In DMD, that missing or incomplete protein is dystrophin. Without functional dystrophin, muscle cells in patients with DMD become fragile, leading to ongoing damage and progressive weakness.
For families affected by nonsense mutation DMD in the United States, the withdrawal means ataluren will not become an available treatment option in the near future. Patients with DMD must continue to rely on currently approved therapies, supportive respiratory and cardiac care and physical therapy to manage symptoms.
PTC Therapeutics, a global biopharmaceutical company focused on rare disorders including DMD, stated that it remains committed to developing and delivering treatments for patients with unmet medical needs.
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