FDA Grants Fast Track Designation for Lexeo’s Gene Therapy for Friedreich’s Ataxia Cardiomyopathy

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Lexeo Therapeutics’ LX2006, an AAV-based gene therapy candidate for the treatment of Friedreich’s ataxia cardiomyopathy.

LX2006 is designed to deliver a functional frataxin gene to promote frataxin protein expression and restore mitochondrial function in myocardial cells.

Fast Track is a process designed to facilitate the development and expedite the review of new drugs intended to treat serious conditions and address unmet medical need. This designation was granted based on available preclinical data. SUNRISE-FA, a phase 1/2 multicenter, 52-week, dose-ascending, open-label clinical trial, is ongoing to evaluate the safety and tolerability, as well as preliminary efficacy, of LX2006 in patients with FA cardiomyopathy.

Friedreich’s ataxia is a rare, genetic, degenerative disorder that affects multiple systems in the body. As the disease progresses, patients typically experience various heart conditions. Hypertrophic cardiomyopathy, fibrosis, heart failure, and arrhythmias are the cause of death in approximately two-thirds of Friedreich’s ataxia patients.

“FA cardiomyopathy is the leading cause of death among FA patients, and there are currently no approved treatment options. The FDA’s Fast Track designation for LX2006 underscores the significant unmet need for effective treatment options to address the cardiac impact of this debilitating disease,” said R. Nolan Townsend, CEO of Lexeo Therapeutics. “We believe today’s Fast Track designation, along with the previously announced Rare Pediatric Disease and Orphan Drug designations granted to LX2006, will allow for enhanced regulatory interactions and the potential for this life-improving therapy to reach FA patients more quickly.”

LX2006 is administered as a one-time intravenous infusion to patients in at least two ascending-dose cohorts with the potential for a third cohort. Long-term safety and efficacy will be evaluated for an additional four years following completion of the initial year of the trial, resulting in data from a total of five years post-LX2006 treatment.

FA cardiomyopathy is the most common cause of mortality in patients with FA affecting approximately 5,000 patients in the United States. LX2006 is designed to target the cardiac manifestations of FA by delivering a functional frataxin gene to promote the expression of the frataxin protein and restore mitochondrial function in myocardial cells. In preclinical studies, LX2006 reversed the cardiac abnormalities in FA disease models and showed improvement in cardiac function and survival while demonstrating a favorable safety profile.

Photo: R. Nolan Townsend, CEO of Lexeo Therapeutics