PTC Therapeutics Faces FDA Rejection On Rare Disease Treatment Over Efficacy Concerns

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The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) on Tuesday related to PTC Therapeutics, Inc.‘s (NASDAQ:PTCT) New Drug Application (NDA) for vatiquinone for Friedreich’s ataxia.

Friedreich’s ataxia (FA) is a rare, inherited neurodegenerative disease that primarily affects the nervous system. It causes progressive spinal cord, peripheral nerves, and cerebellum damage, leading to impaired muscle coordination, difficulty walking, and other neurological symptoms.