PTC to Contest CRL Calling for More Ataluren Trials
PTC Therapeutics received a complete response letter from the FDA for its NDA for ataluren.
The agency said PTC had not produced enough evidence of the drug’s effectiveness for treatment of nonsense mutation dystrophinopathy and called for a further well-controlled clinical trial.
PTC said the agency failed to take the benefit-risk profile into account as well as the high unmet medical need. The company is filing a formal dispute resolution request.