FDA rejects PTC Therapeutics' vatiquinone for Friedreich's ataxia

PTC Therapeutics Inc. (NASDAQ: PTCT) received a Complete Response Letter from the U.S. Food and Drug Administration rejecting its New Drug Application for vatiquinone to treat Friedreich's ataxia in children and adults.

The FDA stated that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support resubmission of the application, according to the company's announcement.

"We are of course disappointed by the FDA's decision to not approve vatiquinone," said Matthew B. Klein, Chief Executive Officer 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."

Vatiquinone is described as a small molecule inhibitor of 15-Lipoxygenase, an enzyme that regulates energetic and oxidative stress pathways disrupted in Friedreich's ataxia. The company states the drug has been evaluated in clinical studies focused on pediatric patients.

Friedreich's ataxia is a rare neuromuscular disorder that affects the central nervous system and heart. The condition causes progressive loss of coordination and muscle strength, leading to balance problems, difficulty speaking and swallowing, spinal curvature, heart conditions, diabetes, and hearing and vision impairment. Approximately 25,000 people have Friedreich's ataxia globally, according to the company.

The Complete Response Letter represents the FDA's determination that the application cannot be approved in its current form. Companies typically respond by conducting additional studies or providing more data to address the agency's concerns.

You May Also Be Interested In

• OpenAI: after the Hugging Face incident, we committed to conducting a much broader review of actions taken by our models
• Bimegren Energy (BESS) files $100M mixed shelf
• Future Money Acquisition gets Nasdaq notice over late 10-Q filing