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news.The US Food and Drug Administration (FDA) has granted rare paediatric disease designation to Vanda Pharmaceuticals’ VCA-894A, an investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S).
CMT2S is a rare, inherited neurological condition that progressively causes motor function loss and muscle weakness. Credit: SkazovD / Shutterstock.com.
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The FDA’s Office of Orphan Products Development and Office of Pediatric Therapeutics granted this status to the therapy.
CMT2S is a rare, inherited neurological condition that progressively causes muscle weakness and motor function loss.
VCA-894A is being investigated specifically for a patient who was diagnosed early in life with this rare subtype of Charcot-Marie-Tooth disease.
The therapy’s target is a specific genetic variant of CMT2S, which to date has not been observed in other patients.
As determined by the FDA, CMT2S predominantly impacts individuals from birth up to 18 years of age, meeting the established criteria for a rare paediatric disease.
The agency noted in its designation letter that severe disease symptoms largely affect paediatric patients.
Vanda Pharmaceuticals president, CEO and chairman Mihael Polymeropoulos said: “CMT2S is a devastating inherited neuropathy for which patients and families have limited treatment options.
“The FDA’s rare paediatric disease designation recognises 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 designation is designed to encourage development of new therapies for serious rare diseases mainly affecting children.
Through this, sponsors may qualify for a priority review voucher if their product achieves approval for a marketing application, although the FDA will determine final eligibility at the time of application review.
Vanda Pharmaceuticals is continuing to progress the development of VCA-894A and is coordinating with the FDA on its clinical programme.
