Enzyvant Begins Rolling Submission for FDA Approval of Tissue-based Therapy for Rare Immune Deficiency

Rare Daily Staff

Enzyvant said that it has started a rolling submission with the U.S. Food and Drug Administration for approval to market its experimental tissue-based therapy RVT-802 to treat the primary immune deficiency resulting from complete DiGeorge Anomaly.

Complete DiGeorge Anomaly (cDGA) is a rare disease affecting approximately 1 in 300,000 infants, or roughly 10 to 20 infants born each year in the United States. Children with cDGA are born without a thymus gland, resulting in severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system.

The condition is fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.
RVT-802 is an experimental tissue-based regenerative therapy designed to treat primary immune deficiency resulting from cDGA. It is designed to be administered only once over the lifetime of the patient.

“This is a milestone not only for Enzyvant, but more importantly for infants with cDGA and their families,” said Alvin Shih, CEO of Enzyvant. “We will continue to press forward with the goal of completing the BLA in 2018 to enable a thorough and timely review by the agency.”

RVT-802 has been granted Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, rare pediatric disease designation, and orphan drug designation by the FDA. As part of the FDA’s commitment to expedite therapeutics that aim to address high unmet medical needs, the agency has agreed to a rolling BLA submission process for therapy.

If you’d like to learn more, you can listen to our 2017 interview with Alvin Shih, CEO of Enzyvant and Louise Markert, the Duke University immunologist who pioneered the therapy.

July 9, 2018
Photo: Alvin Shih, CEO of Enzyvant