FDA Grants Rare Pediatric Disease Status to Vanda’s CMT2S Therapy
Vanda Pharmaceuticals has received Rare Pediatric Disease Designation from the US Food and Drug Administration for VCA-894A, an investigational antisense oligonucleotide therapy being developed for Charcot-Marie-Tooth disease, axonal, type 2S, or CMT2S.
The designation applies to a rare, serious and progressive inherited neurological disorder that causes worsening muscle weakness, sensory impairment and loss of motor function. CMT2S can lead to severe disability, including loss of ambulation, and is estimated to affect fewer than one in one million people globally.
The FDA’s Office of Orphan Products Development and Office of Pediatric Therapeutics granted the designation after determining that CMT2S meets the criteria for a rare pediatric disease. The agency said the condition is serious or life-threatening, primarily affects patients from birth through 18 years of age, and meets the statutory definition of a rare disease.
VCA-894A is being developed for a patient diagnosed early in life with a rare subtype of Charcot-Marie-Tooth disease. According to Vanda, the therapy targets a specific genetic variant associated with CMT2S that has not been observed in any other patient to date.
Charcot-Marie-Tooth disease is a group of inherited disorders that damage peripheral nerves, affecting movement and sensation in the arms, hands, legs and feet. Clinical severity can vary significantly depending on the genetic mutation involved. In CMT2S, progressive nerve dysfunction can result in chronic sensory and motor impairment, reduced mobility and substantial long-term care needs.
Vanda is using an antisense oligonucleotide approach for VCA-894A, a modality designed to alter RNA activity and potentially address disease mechanisms associated with specific genetic variants. The company has not disclosed clinical trial timelines or provided detailed information on the treatment’s mechanism of action.
Mihael H. Polymeropoulos, MD, president, chief executive officer and chairman of Vanda, said the designation highlights the limited treatment options available for patients with the condition.
“CMT2S is a devastating inherited neuropathy for which patients and families have limited treatment options,” Polymeropoulos said. “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.”
The Rare Pediatric Disease Designation is intended to encourage development of therapies for serious or life-threatening rare diseases that predominantly affect children. Sponsors that receive approval for a qualifying product may be eligible for a Rare Pediatric Disease Priority Review Voucher, subject to statutory requirements and FDA review at the time of a marketing application.
A priority review voucher can be used to request accelerated FDA review of a future marketing application or transferred to another company. However, Vanda noted that eligibility for any future voucher will be determined only when a marketing application is reviewed and approved.
Vanda said it will continue advancing VCA-894A and work with the FDA on the development programme for CMT2S.