US pharmaceutical company Vanda Pharmaceuticals Inc (Nasdaq:VNDA) announced on Tuesday that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to VCA-894A, Vanda's investigational antisense oligonucleotide therapy for the treatment of Charcot-Marie-Tooth (CMT) disease, axonal, type 2S (CMT2S), a rare, serious, and progressive inherited neurological disorder.

This 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 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. Vanda says that 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 paediatric disease because it is a serious or life-threatening condition whose manifestations primarily affect individuals from birth to 18 years of age and it meets the statutory definition of a rare disease.

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 says that it is advancing development of its investigational therapy for CMT2S and will continue to work closely with the FDA regarding the development program.