FDA Grants Enzyvant Fast Track and Rare Pediatric Disease Designations for Farber Disease Therapy

FDA Grants Enzyvant Fast Track and Rare Pediatric Disease Designations for Farber Disease Enzyme Replacement Therapy

Photo: Rachelle Jacques, CEO of Enzyvant

Rare Daily Staff

The U.S. Food and Drug Administration granted Enzyvant Rare Pediatric Disease and Fast Track designations for RVT-801, an experimental enzyme replacement therapy for Farber disease, a rare lysosomal storage disorder.

Farber disease is 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 causes inflammation and cell death.

Farber disease patients typically present with 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. Disease onset is usually in early childhood but may occur later in life.

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. In a mouse model of Farber disease, RVT-801 was shown to be biologically active, reducing accumulation of ceramides and related inflammation in tissues.

“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.

“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, which can be used to reduce the FDA review time on a new drug application to six months from ten months.

The vouchers are potentially lucrative because they are transferrable. Most recently, GW Pharmaceuticals sold a priority review voucher for $105 million.

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.

RVT-801 has previously been granted orphan drug designations by the FDA and European Medicines Agency.