FDA Asks Gene Therapy Biotech to Run Clinical Trial That Might Be Impossible

MedCity News February 1, 2023
Frank Vinluan

Taysha Gene Therapies has encouraging data from an open-label, Phase 1/2 study in the rare disease giant axonal neuropathy. It might not be enough. The FDA recommended the biotech conduct a randomized and placebo-controlled study—a challenge in any ultra-rare disease.

One challenge of developing a drug for a disease with no approved therapies is that there isn’t an established clinical trial roadmap to follow. Taysha Gene Therapies hoped the trial design for its gene therapy for a rare neuromuscular disease would put it on the path to a regulatory submission. FDA questions about the study mean that the path forward is almost certainly longer, and might not even be possible.

After meeting with FDA to discuss data from a small,...

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