Enzyvant initiated its rolling submission to the FDA of a BLA for RVT 802 to treat complete DiGeorge Anomaly effects.

Enzyvant, has initiated its rolling submission of a Biologics License Application (BLA) for RVT 802 to the FDA. The company expects to complete its BLA submission by the end of 2018. 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 (RMAT) designation, rare pediatric 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 BLA submission process for RVT-802.

Comment: Complete DiGeorge Anomaly (cDGA) is a rare disease affecting approximately 1 in 300,000 infants, or roughly 10-20 infants born each year in the United States. Children with cDGA are born without a thymus gland, resulting in severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. cDGA is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.