Lexeo to start phase I/II clinical trial for patients with Friedreich’s ataxia cardiomyopathy

May 26, 2022 -- Gene therapy company Lexeo Therapeutics in 2022 plans to launch a phase I/II clinical trial of its adeno-associated virus-based therapy designed to intravenously deliver a functional frataxin gene for the treatment of Friedreich’s ataxia (FA) cardiomyopathy. CEO Nolan Townsend told ScienceBoard.net at the American Society of Gene & Cell Therapy (ASGCT) 2022 annual meeting in Washington, DC that LX2006, whose investigational new drug application was recently cleared by the U.S. Food and Drug Administration, is the first disease-modifying, clinical-stage gene therapy treatment for Friedreich's ataxia and the first clinical-stage program from the company's cardiovascular gene therapy pipeline.

Reata Pharmaceuticals Announces FDA Filing Acceptance and Priority Review Designation for the NDA for Omaveloxolone for the Treatment of Patients with Friedreich’s Ataxia

May 26, 2022. PLANO, Texas--(BUSINESS WIRE)-Today announced that the U.S. Food and Drug Administration (“FDA”) has accepted for filing and granted Priority Review of its New Drug Application (“NDA”) for omaveloxolone for the treatment of patients with Friedreich’s ataxia. The FDA indicated that at this time it has not identified any potential review issues. The NDA is supported by the efficacy and safety data from the MOXIe Part 2 trial and additional supporting data from the MOXIe Part 1 and MOXIe Extension trials. Omaveloxolone received Fast Track Designation in November 2021 and Rare Pediatric Disease Designation in May 2022. The FDA grants Priority Review to medicines that may offer significant improvements in the treatment, diagnosis, or prevention of a serious condition. This Designation shortens the FDA’s review of the NDA to eight months from the time of submission, versus a standard review timeline of 12 months. The FDA has assigned a Prescription Drug User Fee Act (“PDUFA”) target action date of November 30, 2022. The FDA indicated it is currently planning to hold an advisory committee meeting to discuss the application. “We are pleased with the FDA’s decision to grant Priority Review to our NDA for omaveloxolone for the treatment of patients with Friedreich’s ataxia in the United States,” said Warren Huff, Reata’s Chief Executive Officer. “With the FDA’s acceptance of our NDA for filing, omaveloxolone is now one step closer to potentially providing a treatment option for patients with Friedreich’s ataxia, a rare, genetic, debilitating, and degenerative neuromuscular disorder with no approved therapies. We look forward to working with the FDA during the review process, and if approved, we are looking forward to a commercial launch in early 2023.”