CAMBRIDGE, Mass. and BASEL, Switzerland, March 21, 2019 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing and commercializing transformative therapies for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric 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.
“These FDA designations underscore the significant need for a treatment for Farber disease and position RVT-801 to become available to patients as quickly as possible following a successful development program,” said Rachelle Jacques, CEO of Enzyvant.
The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 individuals in the U.S. If a Biologics License Application for RVT-801 is approved, Enzyvant is eligible to receive a priority review voucher.
Fast Track is a process designed to facilitate the development and rapid review of drugs to treat serious conditions and fulfill unmet medical needs. The purpose of the designation is to deliver important new drugs to patients as rapidly as possible.
About Farber Disease
Farber disease is rare and thought to be significantly underdiagnosed, with patients often misdiagnosed with juvenile idiopathic arthritis. The disease is caused by mutations in the ASAH1 gene, resulting in deficiency of acid ceramidase, a critical lysosomal enzyme. This deficiency leads to the accumulation of ceramide within cells, which has pro-inflammatory and pro-apoptotic effects.
Farber disease patients typically present with the cardinal symptoms of joint contractures or arthritis, subcutaneous nodules and/or a weak or hoarse voice. More severe symptoms may also include impaired cognitive development due to brain involvement as well as impact on lungs, liver and bones (osteolysis). Disease onset is usually in early childhood but may occur later in life.
RVT-801 is a recombinant form of human acid ceramidase (rhAC) being developed as a potential enzyme replacement therapy for acid ceramidase deficiency, manifesting as Farber disease. In a mouse model of Farber disease, RVT-801 was shown to be biologically active, reducing accumulation of ceramides and related inflammation in tissues.
Enzyvant, part of Roivant Sciences, is a biopharmaceutical company focused on developing transformative therapies for people with rare diseases. No matter how small the community or complex the condition, we are relentless and purposeful in our efforts to develop and deliver therapies that will bring the greatest benefit to patients with rare diseases and their families. Our current pipeline of therapies is for patients with congenital athymia and Farber disease, and we are working to expand our portfolio to treat other rare diseases and conditions. We are dedicated to rare disease communities because, at Enzyvant, we believe that rare should never mean alone.
For more information, please visit www.enzyvant.com.
About Roivant Sciences
Roivant aims to improve health by rapidly delivering innovative medicines and technologies to patients. We do this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization.
For more information, please visit www.roivant.com.