FDA accepts for filing BLA for RVT 802 to treat pediatric congenital athymia, a condition associated with DiGeorge Anomaly

Enzyvant announced that the FDA has accepted for filing its Biologics License Application (BLA) for RVT 802, a novel investigational tissue-based regenerative therapy designed to treat pediatric congenital athymia, and granted Priority Review.

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 anticipates a regulatory decision in December 2019.

The BLA filing for RVT 802 included clinical data that demonstrated long-term durability of treatment with RVT 802. At the time of the BLA filing, a total of 93 patients received RVT 802 across multiple clinical studies, including 85 patients who met the criteria for inclusion in the efficacy analysis. The Kaplan-Meier estimates of survival [95% confidence interval] at year one and year two post treatment were 76% [66 - 84] and 75% [66 - 83], respectively. For patients surviving 12 months post-treatment, there was a 93% probability of surviving 10 years post-treatment. During clinical development, the most commonly ( greater than 5%) reported RVT 802 related adverse events included thrombocytopenia (11%), neutropenia (8%), pyrexia (5%), and proteinuria (5%).