FDA grants orphan drug designation to RNA therapy for Charcot-Marie-Tooth disease type 1A

The FDA has granted orphan drug designation to DTx-1252, an investigational small interfering RNA therapeutic to treat Charcot-Marie-Tooth disease type 1A.

According to a DTx Pharma press release, the novel therapy is part of the company’s Fatty Acid Ligand Conjugated OligoNucleotides (FALCON) platform, which uses fatty acids to improve biodistribution and cellular uptake of RNA-based therapeutics. In preclinical studies, DTx-1252 was reported to promote remyelination of axons and improve electrophysiological measurements while increasing muscle mass, grip strength, coordination and agility, the company stated.

According to the release, there are currently no approved therapies that target PMP22, the underlying genetic cause of Charcot-Marie-Tooth disease type 1A, a progressive, neuromuscular disease that leads to loss of muscle function and disability.

“Patients living with [Charcot-Marie-Tooth disease type 1A] are in urgent need of treatment and this represents a significant step forward for the patients and the DTx team,” Artie Suckow, PhD, co-founder and CEO of DTx Pharma, said in the release. “DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics. We look forward to the continued development of DTx-1252.”

The FDA provides orphan drug status to drugs and biologics designed to treat, diagnose or prevent rare diseases that affect fewer than 200,000 people in the United States.