Chief Executive Officer of PTC Therapeutics, Matt Klein, M.D., M.S., F.A.C.S., will be joining FARA to discuss the vatiquinone program. Dr. Klein will be providing insights into the ongoing regulatory process and the next steps in advancing vatiquinone toward potential regulatory approval.
PTC Therapeutics Informational Webinar on January 21, 2025
Join the PTC Therapeutics webinar on Jan. 21, 2025, to learn about the vatiquinone program and next steps toward potential regulatory approval.
Register for the Informational Webinar Hosted by PTC Therapeutics on January 21 at Noon ET
Learn More about the Vatiquinone Pipeline Program
Vatiquinone is a small molecule drug that aims to improve mitochondrial and cellular function.
On December 19, 2024, PTC announced the filing of a New Drug Application (NDA) submission for vatiquinone for the treatment of children and adults living with Friedreich Ataxia. The vatiquinone NDA is based on data from the placebo-controlled MOVE-FA study as well as results from two long-term studies including pediatric and adult FA patients. MOVE-FA was a global registration-directed trial of vatiquinone that enrolled 146 pediatric, adolescent, and adult FA patients, the majority of whom were under 18 years of age.
The FDA has 60 days to accept the New Drug Application submission. If the submission for vatiquinone is accepted, the FDA will begin its review of the full data package.