Vanda Pharmaceuticals Gets FDA Rare Pediatric Disease Designation for CMT2S Therapy
Vanda Pharmaceuticals (VNDA) said Tuesday the Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to VCA-894A, its investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare inherited neurological disorder that progressively causes muscle weakness and loss of motor function. The designation was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics, the company said. The company said VCA-894A targets a unique genetic variant of CMT2S not yet observed in any other patient. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, Vanda may become eligible for a priority review voucher upon approval of a qualifying marketing application, with eligibility to be determined at the time of review, according to the company.
Vanda Pharmaceuticals (VNDA) said Tuesday the Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to VCA-894A, its investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), a rare inherited neurological disorder that progressively causes muscle weakness and loss of motor function.
The designation was granted by the FDA's Office of Orphan Products Development and Office of Pediatric Therapeutics, the company said.
The company said VCA-894A targets a unique genetic variant of CMT2S not yet observed in any other patient.
Under the FDA's Rare Pediatric Disease Priority Review Voucher program, Vanda may become eligible for a priority review voucher upon approval of a qualifying marketing application, with eligibility to be determined at the time of review, according to the company.