Posted by Michael Wonder on 21 Mar 2019
Enzyvant's investigational Farber disease enzyme replacement therapy, RVT-801, receives FDA fast track and rare paediatric disease designations
21 March 2019 - Enzyvant today announced that the U.S. FDA has granted are paediatric disease and fast track designations for RVT-801, an investigational enzyme replacement therapy.
RVT-801 has previously been granted orphan drug designations by the FDA and European Medicines Agency and is currently in preclinical development for Farber disease, a rare condition for which there are no approved treatments.
RVT-801 is a recombinant form of human acid ceramidase being developed as a potential enzyme replacement therapy for acid ceramidase deficiency, manifesting as Farber disease.
