RVT-802 is a biologic therapy that was initially developed by Dr. Louise Markert, Professor of Pediatrics at Duke University, 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 75% for treated patients. The findings of Dr. Markert and her research team have been published in the New England Journal of Medicine as well as numerous other peer-reviewed scientific journals and clinical publications.
RVT-802 has been granted orphan drug designation by the Food and Drug Administration (FDA) and Enzyvant anticipates a potential BLA filing in 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.