FDA grants fast track designation to new drug for Friedreich's ataxia cardiomyopathy
Lexeo Therapeutics is one step closer to receiving approval from the U.S. Food and Drug Administration (FDA) for its new drug candidate for Friedreich's ataxia (FA) cardiomyopathy.
The New York City-based pharmaceutical company announced that LX2006, its AAVrh.10hFXN-based gene therapy candidate for patients with FA cardiomyopathy, has been granted the FDA’s fast track designation. This accelerates the review process, helping ensure treatments that address unmet needs get into the hands of healthcare providers as quickly as possible.
FA is a rare genetic disorder that impacts a person’s nervous system. It typically starts showing in patients around the ages of 10 to 15, though patients with late-onset FDA may not develop symptoms until well into their adult years. Cardiomyopathy is a common symptom among patients presenting with FA. There is currently no cure for FA, though a variety of treatments have been developed to manage specific symptoms. LX2006 works by delivering a functional frataxin gene to the patient, promoting frataxin expression and restoring mitochondrial function. The drug previously received the FDA’s orphan drug designation for the same indication.
“FA cardiomyopathy is the leading cause of death among FA patients, and there are currently no approved treatment options,” R. Nolan Townsend, CEO of Lexeo Therapeutics, said in a statement. “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.”
The FDA’s decision was based in part on initial data from SUNRISE-FA, an ongoing clinical trial exploring the drug’s safety and effectiveness in two separate cohorts of patients with FA cardiomyopathy. The open-label trial will follow patients for up to five years following treatment.
A busy year for Lexeo Therapeutics
This news represents the latest of many significant announcements Lexeo Therapeutics has already made in 2024. In February, for instance, the company announced the appointment of a new chief development officer, chief medical officer, senior advisory and chief business and legal officer. One month later, Lexeo Therapeutics closed a funding round worth $95 million that included contributions from both new and existing investors.
Michael has more than 18 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.