VCA-894A

July 7, 2026: Vanda Pharmaceuticals Inc. (NASDAQ: VNDA) announced the FDA granted Rare Pediatric Disease Designation to VCA-894A, its investigational antisense oligonucleotide (ASO) therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare, serious, progressive inherited neuromuscular disorder. The designation was issued by FDA’s Office of Orphan Products Development and Office of Pediatric Therapeutics, with FDA noting that CMT2S’s serious manifestations primarily affect pediatric patients and that the disease meets the statutory definition of a rare disease.

VCA-894A is being developed for a patient with a rare CMT2S subtype; Vanda states the therapeutic target is a unique IGHMBP2 cryptic splice-site variant not yet observed in other patients. The company said it is advancing development and will continue working with FDA on the development program.

Vanda noted that, under the Rare Pediatric Disease Priority Review Voucher program, a sponsor may become eligible for a priority review voucher upon approval of a qualifying marketing application, with eligibility determined at the time of application review and approval.