Experimental Rare Disease Therapy Wins New FDA Reg Med Designation

Last month the U.S. Food and Drug Administration granted Enzyvant both Breakthrough Therapy Designation and the newly established Regenerative Medicine Advanced Therapy Designation for its investigational cell therapy to treat complete DiGeorge Syndrome, a rare and fatal disease. Enzyvant is the first company to win both designations and only the second to win the Regenerative Medicine Advanced Therapy Designation established through the 21st Century Cures Act. The designations provide for accelerated review pathways and special access to developmental guidance from the FDA. We spoke to Alvin Shih, CEO of Enzyvant and Louise Markert, the Duke University immunologist who has been pioneering the therapy, about Complete DiGeorge Syndrome, the innovative approach they are using to treat it, and the path forward for the therapy. As a matter of disclosure, Shih is co-chair of Global Genes Medical and Scientific Advisory Board.