Enzyvant Files Rolling BLA for DiGeorge Anomaly Therapy

Enzyvant Sciences, one of Vivek Ramaswamy’s numerous ‘Vant’ companies, filed a rolling submission of a Biologics License Application (BLA) for RVT-802 with the U.S. Food and Drug Administration (FDA) for the treatment of DiGeorge Anomaly (cDGA), an immunodeficiency disease that impacts infants.

Enzyvant announced the BLA ahead of parent company Roivant Sciences' first Pipeline Day, which was held Tuesday. Enzyvant said it expects to complete the BLA submission by the end of the year.

RVT-802 is an investigational, tissue-based regenerative therapy designed to treat the primary immune deficiency resulting from congenital athymia associated with complete DiGeorge Anomaly. The therapy is expected to be a “one-and-done” treatment. Enzyvant is collaborating with Duke University to advance the development of RVT-802, the company said.

DiGeorge Anomaly is a rare disease affecting approximately one in 300,000 infants, or roughly 10 to 20 infants born each year in the United States, Enzyvant said. 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. If left untreated, complete DiGeorge Anomaly is fatal, typically within the first 24 months of life. RVT-802 has garnered a number of designations from the FDA, including Breakthrough Therapy, Regenerative Medicine Advances Therapy, rare pediatric disease and orphan drug designations.

Alvin Shih, chief executive officer of Enzyvant, said the rolling BLA submission is a milestone for the company, as well as the infants with cDGA. The company aims to complete the BLA this year in order to enable to timely review by the FDA, Shih said.

Days before the company announced it had initiated the rolling BLA, the company formed a partnership with contract research organization Visikol to provide a quantitative technique for assessment of RVT-802. Enzyvant said that will be an important step in ongoing efforts to support an FDA filing.

Ramaswamy praised Enzyvant’s submission and that company’s “dedication to improving the lives of patients suffering from this fatal condition.”

“The ultimate metric of Roivant’s success will be the delivery of truly innovative therapies to patients suffering from serious diseases,” Ramaswamy said in a statement.

In addition to RVT-802, Enzyvant also plans to begin a clinical trial for RVT-801, an investigational enzyme replacement therapy for the treatment of Farber disease. RVT-801 is a recombinant form of human acid ceramidase. The FDA and the European Medicines Agency granted RVT-801 orphan drug designation.