PTC finds itself in quagmire of regulatory setbacks, pushes back timeline on PKU treatment
PTC Therapeutics has delayed its plans by nine months to apply for FDA approval of its treatment for phenylketonuria, a rare disease that causes protein building blocks to accumulate in the body, after the agency asked for a new mouse study.
Originally, PTC wanted to submit its FDA filing for sepiapterin before the end of this year. Now, the agency asked for a half-year mouse study on the drug’s cancer risk. PTC expects it will submit for potential FDA approval in the third quarter of 2024.
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