Genervon’s GM604 Gets Orphan Drug Status

Genervon Biopharmaceuticals’ GM604, an investigational treatment for amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the FDA, positioning it for market perks should the candidate win approval, the California company said Monday.

Genervon is currently conducting a Phase IIa randomized, double-blind study of the drug at two U.S. sites. The study consists of 12 ALS patients ages 18 and over with onset of symptoms within the last two years.

Orphan drug status is granted to drugs that treat conditions with fewer than 200,000 patients in the U.S. Drugmakers developing orphan drugs get seven years of marketing exclusivity and clinical trial tax incentives.

GM604 was also granted FDA fast-track status for ALS last year. Genervon was also recently notified that it will be issued a patent for the drug on March 18. — Lena Freund

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