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Transformative therapies for rare diseases with high unmet needs.
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Pediatric congenital athymia
Children with congenital athymia are generally born without a thymus, resulting in severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. Left untreated, congenital athymia is fatal, typically within the first 24 months of life.
Acid ceramidase deficiency manifesting as Farber disease
Acid ceramidase deficiency is a devastating lysosomal storage disease caused by a mutation in both alleles of the ASAH1 gene, resulting in the deficiency of the lysosomal enzyme acid ceramidase.
June 5, 2019
Enzyvant today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its Biologics License Application (BLA) for RVT-802, a novel investigational tissue-based regenerative therapy designed to treat pediatric congenital athymia, and granted Priority Review.
April 3, 2019
Enzyvant today announced that its investigational regenerative treatment (RVT-802) in late-stage development for congenital athymia will be featured during a platform presentation, at a symposium, and in a poster session at the Clinical Immunology Society (CIS) 2019.
March 21, 2019
The FDA has granted Rare Pediatric Disease and Fast Track designations for RVT-801, an investigational enzyme replacement therapy.