PIPELINE

We are focused on transformative therapies for rare, often fatal, diseases. We are advancing a platform based on two well-characterized technologies with platform potential: T cell generation to treat profound immunodeficiency disorders, and recombinant human acid ceramidase (rhAC) to address the harmful impacts of excess ceramide build-up.

RVT-802

RVT-802 is a novel investigational tissue-based regenerative therapy designed to treat patients with pediatric congenital athymia. Congenital athymia is a rare, and if untreated, deadly condition associated with complete DiGeorge Syndrome (cDGS), CHARGE syndrome, and FOXN1 deficiency.

The U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for RVT-802 and Enzyvant anticipates a regulatory decision in December 2019.

RVT-802 has been granted Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Orphan Drug designations by the FDA, as well as Advanced Therapy Medicinal Product (ATMP) classification and Orphan Drug designation by the European Medicines Agency.

In 2016, Enzyvant entered into an exclusive worldwide licensing agreement with Duke University to develop RVT-802. M. Louise Markert, M.D., Ph.D., Professor of Pediatrics at Duke University School of Medicine, has led research on the treatment of immunodeficiency in patients with congenital athymia.

RVT-801 (rhAC)

RVT-801, a recombinant form of human acid ceramidase (rhAC), is being developed as an enzyme replacement therapy for patients with acid ceramidase deficiency manifesting 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 RVT-801 in patients with Farber disease.

RVT-801 has been granted U.S. Food and Drug Administration (FDA) Rare Pediatric Disease and Fast Track designations as well as Orphan Drug designations by the FDA and European Medicines Agency.

Enzyvant is developing RVT-801 in collaboration with Professor Edward Schuchman, PhD, at the Icahn School of Medicine at Mount Sinai.

Contact us for more information.