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 (CMT) disease known as CMT2S. 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. 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.
"CMT2S is a devastating inherited neuropathy for which patients and families have limited treatment options," said Mihael H. Polymeropoulos, M.D., President, Chief Executive Officer and Chairman of Vanda. "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."
CMT2S is a rare genetic disorder characterized by chronic progressive sensory and motor impairment that can lead to severe disability, including loss of ambulation. The FDA noted in its designation letter that the serious manifestations of the disease primarily affect pediatric patients.
The Rare Pediatric Disease Designation is intended to encourage the development of new therapies for serious or life-threatening rare diseases that primarily affect children. Under the Rare Pediatric Disease Priority Review Voucher program, a sponsor whose product ultimately meets all statutory requirements may become eligible to receive a priority review voucher upon approval of a qualifying marketing application. Eligibility for any future priority review voucher will be determined at the time of a marketing application's review and approval.
Vanda is advancing development of its investigational therapy for CMT2S and will continue to work closely with the FDA regarding the development program.
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