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Vanda Pharmaceuticals (Nasdaq: VNDA) gained after three straight sessions of declines on Friday after it announced that the U.S. Food and Drug Administration had granted orphan drug designation for its experimental treatment VCA-894A as a treatment for a type of rare nerve disorder called Charcot-Marie-Tooth disease (CMT).
VCA-894A is an antisense oligonucleotide targeting immunoglobulin binding protein 2 (IGHMBP2), mutations of which are believed to play a key role in the pathogenesis of a subtype of CMT called CMT type 2S (CMT2S).
The Food and Drug Administration (FDA) has granted Orphan Drug Designation to VCA-894A for CMT2S patients with a variant of IGHMBP2. An inherited neurological condition with no available treatments, CMT2S is estimated to affect less than 1 in 1,000,000 individuals globally.
“This appointment is an important milestone, which we believe may enable the development of individualized therapies tailored to the individual genetic variants of patients with CMT2S,” noted Miel Polymopoulos, CEO of Vanda (VNDA), noted.
In addition to tax credits for clinical trial costs and a user fee waiver for marketing applications, developers of orphan drugs can claim seven years of marketing exclusivity when those drugs are approved.
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