Enzyvant announced that the Food and Drug Administration (FDA) has granted RVT-801 both Rare Pediatric Disease and Fast Track designations for Farber disease.
Farber disease is caused by mutations in the ASAH1 gene, causing a deficiency of the lysosomal enzyme ceramidase. This leads to the accumulation of ceramide within cells, which can result in inflammatory and apoptotic effects. Major symptoms of Farber disease include joint contractures, arthritis, subcutaneous nodules and/or a weak or hoarse voice.
RVT-801 is a recombinant form of human acid ceramidase (rhAC) currently in development for potential enzyme replacement therapy for acid ceramidase deficiency. RVT-801 was shown to be biologically active in a preclinical model of Farber disease, as seen by decreased ceramide accumulation and related tissue inflammation.
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The Rare Pediatric Disease designation is intended for serious or life-threatening diseases that mainly affect children aged ≤18 years and <200,000 individuals in the US. A Fast Track designation can help the development and rapid review of drugs to treat serious conditions and fulfill unmet needs.
For more information visit Enzyvant.com.
This article originally appeared on MPR
