PTC Therapeutics, Inc. has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for vatiquinone, a treatment candidate for Friedreich’s ataxia in both children and adults.
In the CRL, the FDA stated that substantial evidence of efficacy was not demonstrated, and that an additional well-controlled clinical trial would be required to support any future resubmission.
“We are of course disappointed by the FDA’s decision,” said Dr. Matthew B. Klein, CEO of PTC Therapeutics. “We believe the data collected to date show that vatiquinone could offer a safe and effective therapy for people living with Friedreich’s ataxia. We intend to meet with the FDA to explore potential next steps.”
Friedreich’s ataxia is a rare, progressive, and debilitating genetic disorder that affects muscle coordination and can lead to serious complications, including heart disease and diabetes. There are currently limited treatment options available, underscoring the urgency for new therapies.
PTC Therapeutics has not yet announced a timeline for meeting with the FDA or for initiating the additional study requested.