FDA Grants Lexeo Therapeutics Rare Pediatric Disease Designation

Rare Daily Staff

The U.S. Food and Drug Administration granted Lexeo Therapeutics Rare Pediatric Disease designation and Orphan Drug designation to LX2006 for the treatment of Friedreich’s ataxia, a rare neuromuscular disease.

Photo: R. Nolan Townsend, CEO of Lexeo Therapeutics

Friedreich’s ataxia (FA) is a rare, degenerative, multi-system disorder affecting approximately one in 50,000 people in the United States. It is an inherited condition caused by a gene mutation that disrupts the normal production of the protein frataxin, which functions in the mitochondria of the cell. It is inherited in an autosomal recessive manner, usually beginning in childhood and leads to impaired muscle coordination that worsens over time, typically progressing to serious heart conditions that can lead to heart failure, which is the most common cause of death in FA patients.

LX2006 is an IV-administered, adeno-associated virus gene therapy encoding the human frataxin gene. The designations granted to LX2006 cover cardiac disease and broader symptoms associated with FA.

“Being granted both Rare Pediatric Disease and Orphan Drug designation shows the tremendous urgency for new, impactful therapeutic approaches such as LX2006 for people diagnosed with Friedreich’s ataxia,” said R. Nolan Townsend, CEO of Lexeo Therapeutics. “It is critical that we advance new disease-modifying therapies with the potential to transform the lives of FA patients, and we look forward to continuing our collaboration with the FDA as we advance LX2006 through clinical development.”

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 United States. If a biologics license application for LX2006 is approved, Lexeo may be eligible to receive a priority review voucher that may be sold or transferred.

The voucher, which reduces the standard review time for a new drug application by four months, is potentially lucrative because it is transferable. Most recently, Rhythm Pharmaceuticals sold a priority review voucher to Alexion for $100 million.

Lexeo plans to initiate a phase 1/2 clinical trial of LX2006 in patients with cardiomyopathy associated with FA in 2021.