FDA grants Vanda rare pediatric disease tag for CMT2S therapy

Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) announced that the U.S. Food and Drug Administration has granted Rare Pediatric Disease Designation to VCA-894A, its investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S).

The designation was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics. 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 age 18 and meets the statutory definition of a rare disease.

CMT2S is an inherited neuromuscular disorder that progressively leads to muscle weakness and loss of motor function, with an estimated prevalence of less than 1 in 1,000,000 worldwide. The disease can result in significant disability, including loss of ambulation. VCA-894A targets a unique variant of CMT2S not yet observed in any other patient, according to the company.

VCA-894A is a 2'-O-methoxyethyl phosphorothioate oligonucleotide that specifically targets a cryptic splice site variant within IGHMBP2, the gene associated with CMT2S.

"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.

Under the Rare Pediatric Disease Priority Review Voucher program, a sponsor whose product 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.

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