According to a press release from late July 2021, LX1004, a treatment for patients with CLN2 Batten disease, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. The gene therapy, developed by gene therapy company LEXEO Therapeutics (“LEXEO”), is currently in Phase 1/2 clinical testing.
LX1004 for CLN2
An estimated 1,000 patients worldwide have CLN2 Batten disease, also known as late-infantile neuronal ceroid lipofuscinosis (NCL). Typically, NCL is a fatal, inherited nervous system disorder which is characterized by the accumulation of lipopigments (fats and proteins) in bodily tissues. As these build up, it causes brain, retina, and central nervous system (CNL) neuron death. In most cases, Batten disease is fatal by teens or early twenties, depending on the type (infantile, late-infantile, juvenile, adult).
CLN2 is one of the more common forms of Batten disease and results from CLN2 gene mutations. Due to these mutations, patients do not produce enough tripeptidyl peptidase 1 (TPP1) enzyme, preventing cellular waste disposal. CLN2 generally manifests between ages 2-4, with a normal lifespan of 8-12 years. Symptoms include seizures; visual impairment; changes in behavior and personality; myoclonic jerks; motor skill regression; and dementia. Learn more about CLN2 here.
LX1004 received both Orphan Drug and Rare Pediatric Disease designations. This gene therapy works by delivering functional CLN2 straight to the CNS using AAV vectors. Orphan Drug designation is given to drugs or biologics designed to treat patients with a rare condition (affecting under 200,000 Americans). As a benefit, LEXEO receives increased regulatory assistance, fee waivers, tax credits, and 7 years of market exclusivity once approved.
Rare Pediatric Disease designation is similarly granted for drugs or biologics designed to treat under 200,000 pediatric patients (aged 18 or younger). If the Biologics License Application (BLA) is approved, this status could also grant LEXEO Priority Review.
Moving forward, LEXEO hopes to continue evaluating LX1004 in clinical development programs, with some clinical trials occurring in 2022.