On July 7, 2026, Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-894A, an investigational antisense oligonucleotide (ASO) therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). 

CMT2S is an inherited neuromuscular disorder marked by progressive muscle weakness and loss of motor function.  

Vanda notes that the therapy is being developed for a patient whose specific genetic variant has not been documented in any other known case, making this an individualized, or “n-of-1,” treatment approach. 

Rare Pediatric Disease Designation is a regulatory pathway meant to encourage development of therapies for serious diseases that primarily affect children under 18 and does not reflect a determination that a therapy is safe or effective.  

In the future, if Vanda continues development of  VCA-894A and formally applies to the FDA for approval, Vanda could then be awarded a priority review voucher (PRV) following review. 

A PRV is basically a fast-pass. The FDA normally takes about 10 months to review a new drug application, but the PRV cuts that down to about 6 months.  

The FDA won’t decide whether Vanda qualifies for the PRV until much later when they review the finished drug application. This first step in the process is a promising one and if successful offers a new potential option for CMT2S patients in the future.