Regenerative therapy is eligible for FDA priority review, expected to be approved by the end of the year

In the United States, about 20 children with congenital thymus are born each yearBecause there is no thymus at birth, it is impossible to produce T-cells that are resistant to infection and the normal functions necessary to regulate the immune system, which in turn leads to severe immune deficienciesCongenital thymus disorder is associated with complete DiGeorge abnormality (cDGA), CHARGE syndrome and FOXN1 deficiencyDue to the susceptibility to infection, children usually die before the age of 2Currently, the FDA does not have approved treatments for the diseaseRVT-802 is designed as a one-time regenerative therapy for immunodeficiency, designed to replicate the process of "T-cells starting from bone marrow stem cells and then transferring to the thymus" that occurs in a healthy and functional immune system without thymus glandsRVT-802 from the baby thymus tissue is treated and cultured and implanted in the quadriceps of the childChildren's bone marrow stem cells are transferred to implanted tissue products and are trained as immune-active newborn T-cellsWith the recovery of T-cell generation, the immune system can be further restored and the body's ability to fight infectionRVT-802 therapy is based on the work of Professor Mary Louise Markert of Duke UniversityThe findings were published in the New England Journal of Medicine, as well as in many other peer-reviewed scientific journals and clinical journalsIn 2016, Enzyvant signed an exclusive global license agreement with Duke University to develop the RVT-802RVT-802 has been awarded breakthrough therapy by the FDA, regenerative medicine advanced therapy (RMAT) as a rare pediatric disease identification and orphan drug eligibilityEnzyvant's development pipeline (Photo: Enzyvant's official website)The BLA application for RVT-802 is based on clinical trial data that demonstrateits its long-term effectivenessA total of 93 children received RVT-802, of which 85 met the criteria for inclusion in efficacy analysisKaplan-Meier estimated survival rates at 76 per cent (66-84 per cent) and 75 per cent (66-83 per cent) in the first and second years after treatment, respectivelyFor children who survived more than 12 months after treatment, their 10-year survival rate is expected to be 93%"Enzyvant is proud to promote rvT-802 as a bold, revolutionary regenerative therapy," said Rachelle Jacques, ceo of Enzyvant,The sense of urgency to save the lives of infants and young children with congenital thymus-free disease has led us to partner with Duke University and continue to inspire us to move quickly into the future with a focused attitudeRVT-802 is a long-term data for disposable therapies that reinforce the potential value of saving and sustaining life protection"
References:Retrieved June, 5, 2019, from https://Retrieved June, 5, 2019, from https://mp.weixin.qq.com/s/duiO_UZzd9wKt05Sq5-YbQRetrieved June, 5, 2019, from https://mp.weixin.qq.com/s/qIebJYFc_GJh73WUz4GWIA