In previous years, PTC Therapeutics has attempted to win regulatory approval for its Duchenne Muscular Dystrophy drug, Translarna (ataluren), but the U.S. Food and Drug Administration has rejected the attempts. The company believes it has data that will support another filing for possible approval.
Translarna, which has been repeatedly authorized for use in Europe and other countries, was rejected for a third time in October 2017. Despite those failures, PTC Therapeutics remained steadfast in its belief in the potential of the drug for DMD patients. The drug is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
Translarna enables the production of full-length, functional dystrophin, the company said. Five years ago, in its Complete Response Letter, the FDA called for additional efficacy data from another trial.
Tuesday morning, the company presented new topline results from Study 041 that show positive benefits for patients with nonsense mutation DMD, a subset of the disease that impacts between 10% and 15% of patients. This mutation creates a premature stop codon in the dystrophin mRNA, which prevents the production of full-length, functional dystrophin protein.
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