RARE Daily

FDA Grants Rare Pediatric Disease Designation to Lexeo Therapeutics CLN2 Batten Disease Therapy

July 28, 2021

The U.S. Food and Drug Administration granted Lexeo Therapeutics Rare Pediatric Disease designation and Orphan Drug designation for LX1004 for the treatment of CLN2 Batten disease, a fatal genetic condition of early childhood.

Photo: R. Nolan Townsend, CEO of Lexeo Therapeutics

CLN2 Batten disease is a rare, neurodegenerative lysosomal storage disorder caused by a deficiency in the enzyme TPP1, which results in accumulation of metabolic waste material and degeneration of tissues including the brain and retina. CLN2 disease is characterized by seizures, rapid deterioration of language and motor functions, cognitive decline, loss of vision and blindness, and premature death by mid-childhood. Current treatment options include palliative care or enzyme replacement therapy, which is administered into the brain through a permanently implanted device on a biweekly basis.

LX1004 is an AAV-mediated gene therapy delivering CLN2 to the central nervous system. It is Lexeo’s lead experimental therapy. The company has completed a phase I clinical trial of LX1004 and plans to advance the program into pivotal studies in 2022. 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 United States. Programs with Orphan Drug status are eligible for various development incentives.

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 (BLA) for LX1004 is approved, Lexeo may be eligible to receive a priority review voucher that may be sold or transferred.

“Both designations granted to LX1004 underscore the critical importance and urgency to advance new treatment approaches for CLN2 Batten disease, a fatal genetic disorder affecting the central nervous system (CNS),” said R. Nolan Townsend, CEO of Lexeo Therapeutics. “We are encouraged by the phase I study results of LX1004, which reached clinical proof of concept, and we look forward to advancing the program through further clinical development with the hope of making it available for patients as soon as possible.”

Author: Rare Daily Staff

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