Enzyvant therapy gets RMAT, breakthrough designations

Enzyvant Sciences Ltd. (Basel, Switzerland) said FDA granted regenerative medicine advanced therapy and breakthrough therapy designations to RVT-802 for complete DiGeorge syndrome (cDGS). The RMAT designation, created by the 21st Century Cures Act, confers the same expedited development and regulatory review actions afforded drug candidates that receive breakthrough therapy designation (see BioCentury Extra, Jan. 24).

RVT-802 consists of cultured, partially T cell-depleted, allogeneic thymic tissue for transplantation. It also has Orphan Drug designation in the U.S. for primary immune deficiency resulting from athymia associated with cDGS. Enzyvant said it plans to submit a BLA to FDA in 1H18 for the indication. The company has exclusive, worldwide rights to RVT-802 from Duke University (Durham, N.C.)...