Vanda Pharmaceuticals Receives FDA Rare Pediatric Disease Designation for VCA-894A

The company states: "Vanda Pharmaceuticals announced that the FDA has granted Rare Pediatric Disease Designation to VCA-894A, Vanda's investigational antisense oligonucleotide therapy for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare, serious, and progressive inherited neurological disorder. The designation was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics. VCA-894A is being developed for a patient who was first diagnosed at an early age with a rare subtype of Charcot-Marie-Tooth disease known as CMT2S.1 CMT2S is an inherited neuromuscular disorder that progressively leads to muscle weakness and loss of motor function, and has an estimated prevalence of less than 1 in 1,000,000 worldwide.2 VCA-894A's therapeutic target is a unique variant of CMT2S not yet observed in any other patient. The severity and clinical presentations of CMT2S are influenced by the diverse genetic variants associated with CMT disease. The FDA determined that CMT2S qualifies as a rare pediatric disease because it is a serious or life-threatening condition whose manifestations primarily affect individuals from birth through 18 years of age and it meets the statutory definition of a rare disease."