2023-06-02 15:15:50 ET
Vanda Pharmaceuticals ( NASDAQ: VNDA ) gained after three straight sessions of declines on Friday after announcing that the FDA granted Orphan Drug Designation for its experimental therapy VCA-894A as a treatment for a type of rare nerve disorder called Charcot-Marie-Tooth disease (CMT).
VCA-894A is an antisense oligonucleotide that targets immunoglobulin mu-binding protein 2 (IGHMBP2), mutations of which are thought to play a key role in causing a subtype of CMT called CMT type 2S (CMT2S).
The FDA has awarded Orphan Drug Designation for VCA-894A for CMT2S patients with a variant of IGHMBP2. An inherited neurological condition with no available treatments, CMT2S is estimated to impact less than 1 in 1,000,000 individuals globally.
"This designation is an important milestone, which we believe may enable the development of individualized treatments tailored to one's genetic variants for patients with CMT2S," CEO of Vanda ( VNDA ) Mihael Polymeropoulos noted.
In addition to tax credits for clinical trial costs and waiver of the user fee for marketing applications, the developers of orphan drugs can claim seven years of marketing exclusivity when those drugs are approved.
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Vanda gains on FDA orphan drug designation for nerve disorder candidate