神经分泌弗里德赖希共济失调资产被FDA授予孤儿地位

2026-05-08 00:54

The US FDA has granted orphan drug designation to Neurocrine Biosciences' (NBIX) NBIB-223 (frataxin) for the treatment of Friedreich ataxia.
Therapies with orphan status have a potential seven years of exclusivity following approval, tax credits for clinical trial expenses, and an exemption from the FDA's prescription drug user fee requirement.
NBIB-223 is a gene therapy that uses Voyager Therapeutics' (VYGR) modified AAV capsid to deliver the frataxin gene throughout the body. The latter company has an option for 40% of US rights.
The only approved therapy for Friedreich ataxia is Biogen's (BIIB) Skyclarys (omaveloxolone).

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