Posted by Michael Wonder on 09 Jul 2018
Enzyvant announces initiation of RVT-802 rolling BLA submission for the treatment of complete DiGeorge anomaly
9 July 2018 - Completion of rolling BLA submission anticipated by end of 2018.
Enzyvant announced today that it has initiated its rolling submission of a biologics license application for RVT-802 to the U.S. FDA.
RVT-802 is an investigational, tissue-based regenerative therapy designed to treat the primary immune deficiency resulting from congenital athymia associated with complete DiGeorge Anomaly (cDGA). RVT-802 is designed to be administered only once over the lifetime of the patient. cDGA is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.
RVT-802 has been granted breakthrough therapy designation, regenerative medicine advanced therapy designation, rare paediatric disease designation, and orphan drug designation by the FDA. As part of the FDA’s commitment to expedite therapeutics that aim to address high unmet medical needs, the Agency has agreed to a rolling submission process for RVT-802.
