PTC Therapeutics, Inc. (PTCT) Faces FDA Setback on Vatiquinone for Friedreich's Ataxia

We recently compiled a list of the 11 Cheap Biotech Stocks to Buy According to Hedge Funds. PTC Therapeutics, Inc. is one of them.

PTC Therapeutics, Inc. (NASDAQ:PTCT) is a biotechnology company focused on developing treatments for rare disorders, including Duchenne muscular dystrophy, AADC deficiency, and phenylketonuria (PKU), with an active pipeline in rare disease research such as Huntington’s disease.

In August 2025, PTCT received FDA approval for Sephience, an oral treatment for PKU, marking a major growth driver. The company’s Q2 revenues totaled $179 million, surpassing expectations, while cash reserves of nearly $2 billion provide financial flexibility for future development.

However, PTC Therapeutics, Inc. (NASDAQ:PTCT) faced a setback with the FDA issuing a Complete Response Letter for vatiquinone, its oral therapy for Friedreich’s ataxia, citing insufficient efficacy evidence and requesting additional studies before resubmission. Management remains committed to continuing clinical development and engagement with regulators.

Strategically, PTC Therapeutics, Inc. (NASDAQ:PTCT) leverages partnerships with major pharma companies like Roche and Novartis to advance its rare disease pipeline. Analysts remain positive on long-term prospects, with average price targets around $68, reflecting confidence in Sephience’s growth potential and ongoing pipeline developments.

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