The Charcot-Marie-Tooth Association (CMTA) is pleased to share exciting news from our CMTA-STAR Alliance Partner, Applied Therapeutics. The company plans to submit a New Drug Application (NDA) to the FDA for govorestat to treat CMT-SORD (aka SORD Deficiency), a significant milestone for the community as it could provide the first regulatory approval in the CMT space.
In July 2024, Applied Therapeutics met with the FDA to discuss the path forward for govorestat. The FDA confirmed that the data gathered thus far from the company’s ongoing INSPIRE Phase III clinical trial for CMT-SORD supports a potential NDA submission under the Accelerated Approval Program. This program allows drugs for severe conditions that fill an unmet medical need, such as CMT, to be approved more quickly based on early evidence, such as a laboratory test result, that predicts a real clinical benefit. Participants in the ongoing INSPIRE study will be offered open-label treatment, meaning every participant will receive the drug. This continuation is crucial as it provides ongoing access to the treatment while participants remain closely monitored for safety. An additional confirmatory study will be required after the drug is approved.
“Today’s announcement from our Alliance Partner Applied Therapeutics is a beacon of hope for the CMT community,” said Sue Bruhn, PhD, CMTA CEO. “The CMTA’s collaborative approach and STAR research investments from gene discovery through clinical trial readiness have been instrumental in achieving this key development milestone in record time, and we remain committed to supporting Applied Therapeutics in its mission to bring this first-ever vital treatment to the CMT-SORD community.”
Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics, said, “We are incredibly pleased to share our progress with the FDA regarding a potential NDA submission for govorestat for the treatment of SORD Deficiency. At Applied, we are dedicated to creating transformative treatments for rare diseases, and we continue to work closely with regulatory agencies and patient advocacy groups to ensure that treatments become available for patients with these debilitating diseases.”
With funding support from CMTA, the SORD gene was discovered in 2020 by a team of researchers at the GENESIS project. Additional investments and CMT-STAR initiatives across research and development, including our partnership with the Inherited Neuropathy Consortium (INC), have supported the development of multiple clinical outcome measures. This commitment to the clinical trial ecosystem has enabled the rapid advancement from gene discovery to the INSPIRE Phase III clinical trial in just two years. We celebrate this encouraging news from our Alliance Partner, Applied Therapeutics, and are proud of the collaborative efforts that have brought us to this point.
Published: August 7, 2024