FDA Rejects PTC Therapeutics’ Friedreich’s Ataxia Drug

The FDA has rejected PTC Therapeutics’ vatiquinone, the corporate’s proposed remedy for Friedreich’s ataxia in youngsters and adults.

In its full response letter, the FDA acknowledged that “substantial proof of efficacy was not demonstrated” and that PTC would wish a further “satisfactory and well-controlled research” to assist a resubmission, the corporate revealed in its press launch Tuesday morning.

Shares of PTC are up about 6% in Tuesday morning buying and selling.

Matthew Klein, the corporate’s CEO, mentioned in a press release that PTC is planning to satisfy with the FDA to debate potential subsequent steps.

Friedreich’s ataxia is a uncommon neuromuscular dysfunction, stemming from a mutation within the frataxin gene that results in mitochondrial dysfunction and cell demise. Signs embody lack of coordination and muscle energy, in addition to issue talking, swallowing and respiratory. In accordance with PTC, about 25,000 individuals have Friedreich’s ataxia globally.

Vataquinone is a small molecule that blocks a few of the mobile pathways that go awry in sufferers with frataxin mutations. The drug had beforehand missed the mark in a Section III, registration-directed trial again in 2023, failing to satisfy its major endpoint of enhancing gait, stability and limb perform after 72 weeks. Nonetheless, based mostly on assembly some secondary endpoints like enhancing stability, PTC went forward and sought registration with the FDA.

The one drug in the marketplace for Friedreich’s ataxia is Skyclarys, accredited in early 2023 when it was held by Reata Prescription drugs. Skyclarys, additionally a small molecule remedy, works by activating different mobile pathways to scale back the irritation that Friedreich’s ataxia could cause. Following the drug’s approval, Biogen acquired Reata for about $7.3 billion.

PTC is the newest biotech to obtain an FDA rejection within the uncommon illness house in current weeks. Final month, Capricor Therapeutics was hit with a shock CRL for its Duchenne muscular dystrophy asset deramiocel, whereas Ultragenyx’s UX111 for Sanfilippo syndrome kind A additionally obtained turned away unexpectedly by the company.