Already on Fast Track, Aceragen Launches with $35 Million and Farber Disease Drug

Published: May 03, 2021
By Kate Goodwin
Grabbing a fast-tracked novel enzyme replacement therapy for a rare pediatric disease, new biopharma Aceragen launched with $35 million in hand for product financing.  
The drug the new company picked up comes from Enzyvant, one of the five Vants entrepreneur Vivek Ramaswamy
 sold off to Japan’s Sumitomo Dainippon Pharma in 2019. The company had already done some FDA legwork for the drug, RVT-801, by gaining Rare Pediatric Disease and Fast Track Designations for it, in addition to Orphan Drug designations by both the FDA and EMA. 
RVT-801 is being developed for Farber disease, an ultra-rare condition typically onset during early infancy. Patients with mild form typically live to only 5-7 years of age, even less in rapid progressive cases. Excess amounts of lipids build up to harmful levels in joints, tissues and the CNS causing severe inflammation with life-threatening complications. There is currently no treatment for the inherited metabolic disorder.  

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