FDA Grants Vanda Rare Pediatric Disease Designation for CMT Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has granted Vanda Pharmaceuticals Rare Pediatric Disease designation for its experimental therapy, VCA-894A, for the treatment of Charcot-Marie-Tooth disease type 2S, a rare nerve disorder that primarily affects children.

The designation is intended to encourage the development of treatments for serious or life-threatening diseases that affect small numbers of pediatric patients.

Charcot-Marie-Tooth disease type 2S (CMT2S) is an inherited neurological condition that damages peripheral nerves, leading to progressive muscle weakness, loss of motor function, and, in severe cases, an inability to walk. The disease has no approved treatments.

VCA-894A is an antisense oligonucleotide that targets a specific mutation in the IGHMBP2 gene believed to drive the disease. The therapy is being developed for a unique genetic variant identified in a single patient, highlighting the growing role of highly personalized medicines.

The FDA’s designation recognizes both the severity of the disease and the lack of available treatment options. It also makes Vanda eligible, if the drug is eventually approved, to receive a priority review voucher. The vouchers can be used to shorten the regulatory review of an application for marketing approval to six from 10 months. The vouchers are potentially lucrative because they are transferrable. Rocket Pharmaceuticals in June sold its Rare Pediatric Disease Voucher for $180 million.