Therapy for a Type of Primary Immune Deficiency Wins Rare Pediatric Disease Designation

Cell-based therapy harvests, cultures, and applies allogeneic thymic tissue

The FDA has granted a rare pediatric disease designation to RVT-802 (Enzyvant), an investigational tissue-based therapy for the treatment of primary immune deficiency resulting from congenital athymia associated with complete DiGeorge syndrome (cDGS). RVT-802 previously received breakthrough therapy, regenerative medicine advanced therapy, and orphan drug designations from the FDA.

cDGS represents approximately 1% of all patients with DiGeorge syndrome, a congenital condition. The three main characteristics of cDGS are congenital heart disease, hypoparathyroidism (leading to low calcium levels), and athymia (lack of thymus tissue). Athymia leads to severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. cDGS is uniformly fatal if untreated, with death typically occurring in the first two years of life due to susceptibility to infection.

RVT-802 is an investigational cell-based therapy using proprietary processes to harvest, culture, and apply allogeneic thymic tissue for the treatment of primary immune deficiency associated with cDGS. RVT-802 is investigational and not approved for commercial use by the FDA or other health regulators at this time.

The FDA grants rare pediatric disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals younger than 18 years of age. With this designation, RVT-802 will be eligible to receive a rare pediatric disease priority review voucher upon biologics license application approval, which Enzyvant anticipates filing in the first half of 2018.

Source: Enzyvant; September 5, 2017.