FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful

The FDA has rejected PTC Therapeutics' application for vatiquinone, a drug intended to treat Friedreich's ataxia, citing insufficient evidence of efficacy135.

The agency issued a Complete Response Letter (CRL), stating that PTC must conduct another adequate and well-controlled study to support future resubmission35.

In the pivotal phase 3 MOVE-FA trial, vatiquinone failed to meet its primary endpoint of a statistically significant change in the modified Friedreich Ataxia Rating Scale (mFARS) score at 72 weeks135.

PTC highlighted positive results in a pre-specified secondary endpoint (the mFARS upright stability subscale) but this was insufficient for FDA approval13.

Company CEO Matthew Klein expressed disappointment but indicated plans to meet with the FDA to discuss potential next steps35.

Analysts noted that the outcome was not surprising, given that PTC was not in advanced label discussions with the agency before the rejection1.

Friedreich's ataxia is a rare, inherited neurodegenerative disease that causes progressive damage to the nervous system, leading to motor coordination difficulties and reduced life expectancy35.

Sources:

1. https://www.biopharmadive.com/news/ptc-drug-for-friedrichs-ataxia-rejected-lilly-sells-nearly-7b-in-bonds/757979/

3. https://www.fiercebiotech.com/biotech/ptc-therapeutics-friedreichs-ataxia-hopes-derailed-crl-fda

5. https://www.benzinga.com/news/fda/25/08/47212794/ptc-therapeutics-faces-fda-rejection-on-rare-disease-treatment-over-efficacy-concerns