The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for RVT-802 (Enzyvant), an investigational tissue-based regenerative therapy for pediatric congenital athymia.

RVT-802, derived from infant thymus tissue, is designed to reconstitute the immune system for the treatment of T cell immunodeficiency resulting from pediatric congenital athymia. The product is implanted into a patient’s quadricep muscle, where bone marrow stem cells migrate to and eventually become naïve, immunocompetent T cells.

The BLA was supported by clinical data that showed the long-term durability of treatment with RVT-802. The data included a total of 93 patients who received RVT-802, including 85 patients who met the inclusion criteria in the efficacy analysis.

Results showed that the Kaplan-Meier estimates of survival [95% confidence interval] following treatment were 76% [66-84%] at year 1 and 75% [66-83%] at year 2. In addition, for patients surviving 12 months after treatment, the researchers calculated a 93% probability of survival 10 years post-treatment. Regarding safety, the most common adverse reactions associated with treatment were thrombocytopenia (11%), neutropenia (8%), pyrexia (5%), and proteinuria (5%).

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“The long-term data for RVT-802 as a one-time treatment reinforces the potentially life-saving value and durable impact of this therapy,” said Rachelle Jacques, Chief Executive Officer of Enzyvant. “We are committed to working collaboratively with payers to establish a value-based reimbursement model that accelerates access for patients.”

RVT-802 has been granted Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Orphan Drug designations by the FDA.

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