RVT-802 is an investigational thymic-tissue based regenerative therapy that is designed to reconstitute the immune system for the treatment of primary immune deficiency resulting from congenital athymia associated with complete DiGeorge Anomaly (cDGA), or forkhead box protein N1 (FOXN1) deficiency. This is a fatal disorder with no approved therapies.
RVT-802 is the first program to be granted both Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy by the FDA.
RVT-801 is a recombinant form of human acid ceramidase (rhAC) for use as an enzyme replacement therapy in acid ceramidase deficiency (ACD), which manifests as Farber disease. Enzyvant is conducting a natural history study of patients with Farber disease to better define the natural course of disease and the relationship between specific symptoms, biomarkers, and prognosis. Enzyvant is also conducting preclinical studies to enable a clinical trial of (rhAC) in patients with Farber disease.
Orphan drug designation for RVT-801 has been granted by regulatory agencies in the United States and the European Union.
Enzyvant is developing this technology in collaboration with Professor Edward Schuchman, PhD, at the Icahn School of Medicine at Mount Sinai.
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