The Food and Drug Administration (FDA) has granted Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations for the investigational tissue-based therapy, RVT-802 (Enzyvant), for the treatment of complete DiGeorge Syndrome (cDGS).

Complete DiGeorge Syndrome is a rare, genetic, congenital disease caused by a defect in multiple genes on chromosome 22. It is mainly characterized by congenital heart disease, hypoparathyroidism, and athymia leading to severe immunodeficiency due to the inability to produce normally functioning T-cells. If left untreated, cDGS is fatal, with death occurring in the first two years of life due to susceptibility to infection. 

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Enzyvant is the first company to announce the simultaneous receipt of RMAT and Breakthrough Therapy designations for an investigational drug. RMAT designation is a component of the 21st Century Cures Act, signed into law in December 2016. To receive this designation, eligible drugs (eg, cell therapies, therapeutic tissue engineering products, human cell and tissue products, or combination of such therapies/products) must be intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and have preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such disease or condition.

RVT-802 is a biologic therapy using proprietary processes to harvest, culture, and apply allogeneic thymic tissue for the treatment of primary immune deficiency resulting from cDGS. The company anticipates a potential BLA filing in the first half of 2018.

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