FDA accepts BLA for Enzyvant's regenerative therapy RVT-802

The US Food and Drug Administration (FDA) has accepted for filing the Biologics License Application (BLA) for RVT-802, a novel investigational tissue-based regenerative therapy designed to treat pediatric congenital athymia, and granted Priority Review, from US biopharma Enzyvant, a member of the Roivant family of companies.

Congenital athymia is a rare and deadly condition associated with complete DiGeorge Anomaly (cDGA), CHARGE syndrome, and FOXN1 deficiency. At this time, the FDA is not planning to hold an Advisory Committee meeting to discuss the application, and Enzyvant says it anticipates a regulatory decision in December 2019.

Around 20 infants are born each year in the USA with congenital athymia, which is fatal if untreated. Death typically occurs in the first 24 months of life due to susceptibility to infection. Currently, there are no FDA-approved therapies for this condition. RVT-802 stimulates and facilitates the body’s production of naive, immunocompetent T cells, with the goal of bolstering the immune system and restoring the body’s ability to fight infection. Investigational RVT-802 is designed to be administered as a single treatment.