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-based milestones up to $226 million, as well as tiered royalties on net sales. In addition, Aceragen entered into a $35 million product financing agreement with NovaQuest Capital Management to fund the development program into a potential registrational study.