RVT-802 is an investigational biologic therapy using proprietary processes to harvest, culture, and apply allogeneic thymic tissue for the treatment of primary immune deficiency resulting from cDGS. Preliminary clinical results suggest a survival rate of over 70% for treated patients.
RVT-802 has been granted Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and orphan drug designation by the FDA. Enzyvant anticipates a potential BLA filing in the first half of 2018.
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.