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

The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) on Tuesday related to PTC Therapeutics, Inc.‘s 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.

Also Read: Sephience Gets FDA Approval, Now A Key Revenue Driver For PTC Therapeutics

“We are of course disappointed by the FDA’s decision to not approve vatiquinone,” said Matthew Klein, CEO of PTC Therapeutics. “We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich’s ataxia. We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL.”

The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission.

In 2023, PTC Therapeutics’ topline results from the MOVE-FA trial of vatiquinone in patients with Friedreich ataxia did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population.

The FARS scale is a series of physical examination assessments to measure disease progression.

The company said that vatiquinone treatment demonstrated significant benefits for key disease subscales and secondary endpoints. 

However, in October 2024,  PTC Therapeutics said the pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression.

Analysis of the MOVE-FA long-term extension study demonstrated that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit (p<0.0001, N=70) on the modified Friedreich Ataxia Rating Scale (mFARS) relative to a matched natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Measures) disease registry.

This treatment difference on the primary endpoint represents a clinically meaningful 50% slowing in disease progression over 3 years.

These results confirm that the slowing of disease progression recorded in the 72-week placebo-controlled MOVE-FA trial is maintained over 144 weeks of treatment.

In February 2025, the FDA accepted the vatiquinone application under Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of August 19.

The company submitted the application in December 2024.

PTCT Price Action: PTC Therapeutics shares were up 4.89% at $52.23 at the time of publication on Tuesday, according to Benzinga Pro data.

Read Next:

• Peabody Energy Stock Surges After Scrapping $3.8 Billion Anglo American Coal Deal

Photo: Shutterstock