Vanda Pharmaceuticals (NASDAQ: VNDA) has announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to its investigational antisense oligonucleotide therapy—VCA-894A—for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S).
CMT2S is a rare, serious, and progressive inherited neurological disorder primarily affecting individuals from birth through 18 years of age.
In a statement, Mihael H. Polymeropoulos, MD, president, CEO and chairman of Vanda, commented, “CMT2S is a devastating inherited neuropathy for which patients and families have limited treatment options. The FDA’s Rare Pediatric Disease Designation recognizes the significant unmet medical need in this patient population and supports our efforts to develop a potentially transformative therapy for patients affected by this rare disease.”
