M2 PHARMA-June 7, 2019-US FDA Accepts Enzyvant Biologics License Application and Priority Review Status for RVT-802, a Novel Investigational Tissue-Based Regenerative Therapy for Pediatric Congenital Athymia
- The US Food and Drug Administration has accepted for filing US-based biopharmaceutical company Enzyvant's Biologics License Application for RVT-802, a novel investigational tissue-based regenerative therapy designed to treat pediatric congenital athymia, and granted Priority Review, the company said.
Congenital athymia is a rare and deadly condition associated with complete DiGeorge Anomaly, CHARGE syndrome, and FOXN1 deficiency.
Children with congenital athymia are born without a thymus, resulting in a severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system.
Approximately 20 infants are born each year in the United States with congenital athymia, which is fatal if untreated.
RVT-802 is a novel investigational tissue-based regenerative therapy designed to treat primary immune deficiency resulting from pediatric congenital athymia. In a healthy, functioning immune system, T cells that start as stem cells in bone marrow become fully developed in the thymus.
Although a causal relationship between acquired athymia
and immune dysregulation has yet to be established, thymectomies in patients younger than 3 months have been shown to result in lower numbers of circulating T cells and diminished responses to T-cell mitogens. Hence, preoperative imaging studies are recommended to verify the presence of mediastinal thymic tissue prior to surgical neck exploration.
RVT-802 is an investigational, single administration, tissue-based regenerative therapy designed to treat the primary immune deficiency resulting from congenital athymia
associated with complete DiGeorge Anomaly (cDGA) which is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.