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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