VNDA News & Events

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."

Vanda Pharmaceuticals announced that Japan's Ministry of Health, Labour and Welfare has granted orphan drug designation to imsidolimab, a high-affinity humanized immunoglobulin G4 monoclonal antibody, for the treatment of generalized pustular psoriasis. The MHLW grants orphan drug designation to medicines intended to treat rare diseases with significant unmet medical need. In Japan, approximately 2,200 people have been diagnosed with GPP. In Japan, orphan drug designation provides development incentives, including subsidies for research and development costs, as well as the potential for up to 10 years of market exclusivity following approval. This designation follows similar regulatory recognition in the U.S.