LEXEO Therapeutics Receives Rare Pediatric Disease Designation and Orphan Drug Designation for LX2006 for the Treatment of Friedreich’s Ataxia

 LEXEO Therapeutics, a clinical-stage gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation and Orphan Drug designation to LX2006 for the treatment of Friedreich’s ataxia (FA). LX2006 is an IV-administered, adeno-associated virus (AAV)-mediated gene therapy encoding the human frataxin gene. The designations granted to LX2006 cover cardiac disease and broader symptoms associated with FA.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S. If a biologics license application (BLA) for LX2006 is approved, LEXEO may be eligible to receive a priority review voucher that may be sold or transferred.

The Orphan Drug designation is granted by the FDA to drugs or biologics intended to treat a rare disease that affects fewer than 200,000 people in the U.S. Programs with Orphan Drug status are eligible for various development incentives.

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