FDA grants Enzyvant's RVT-801 rare pediatric disease eligibility and fast-track eligibility

Farber disease is a rare and considered significantly underdiagnosed disease, often misdiagnosed as juvenile isopathic arthritisThe disease is caused by mutations in the ASAH1 gene, which cause the deficiency of acidic ceramidase, an important lysosome enzyme, Enzyvant(http://announced that the u.SFood andDrug(http://Administration (
FDA(http://has granted RVT-801 for rare pediatric diseases and fast-track eligibilityRVT-801 is an experimental enzyme replacement therapy (ERT) that is currently in preclinical development and is being developed for treatment of Farber disease, a rare solyase memory disorder that causes a variety of symptoms throughout the body and has not yet been approved as a treatmentRVT-801 is a recombinant human acidic ceramidase (rhAC) as a potential enzyme replacement therapy for acidic ceramidase deficiency, manifested in favelassisIn the Farwell mouse model, RVT-801 was shown to be biologically active, reducing the accumulation of ceramide and associated tissue inflammationThe FDA's rare pediatric disease eligibility is granted to those developed for serious or life-threatening diseases that affect the u.Sunder the age of 18 or younger and have a number of under 200,000 diseases If the RVT-801's Biologics Licensing Application (BLA) is approved, Enzyvant will be eligible for a Priority Review Certificate (PRV) 　　Fast-track eligibility is designed to accelerate drug development and rapid review for serious diseases to address the needs of severely unmet medical (http:// in key areas The fast-track eligibility of experimental drugs means that drug companies can interact more frequently with the FDA during the development phase and may also be given priority review by the FDA after filing a listing application