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
Published: May 03, 2021
Acquires worldwide rights to RVT-801, a novel enzyme replacement therapy for treatment of Farber disease
NovaQuest investment to fund product and clinical development
Protocol for potential single registration study reviewed with FDA and EMA
Program has been granted rare pediatric disease status, fast track and orphan designations, and is eligible for a priority review voucher once approved
RESEARCH TRIANGLE PARK, N.C. & BASEL, Switzerland (BUSINESS WIRE)
Aceragen, Inc., a biopharmaceutical company focused on advancing transformational therapeutics for rare and ultra-rare diseases, today announced the acquisition of Enzyvant’s RVT-801 (now ACG-801), an investigational enzyme replacement therapy (ERT) for acid ceramidase deficiency presenting as Farber disease, a lysosomal storage disease with a unique, severe inflammatory phenotype for which no disease-specific therapy exists. Enzyvant will receive an upfront payment and development and sales-ba