FDA Grants Rare Pediatric Disease Designation to RVT-802

Mathew Shanley

In conjunction with the introduction of former Spark Therapeutics Head of Commercial Morgan Malloy as the company’s new Chief Commercial Officer, biopharmaceutical company Enzyvant has announced the U.S. Food and Drug Administration (FDA) Rare Pediatric Disease designation of RVT-802.

RVT-802, an investigational tissue-based therapy, is intended for the treatment of primary immune deficiencies resulting from congenital athymia associated with complete DiGeorge Syndrome.

Previously, the FDA has granted RVT-802 with Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and orphan drug designation.

Complete DiGeorge syndrome (cDGS) represents an estimated 1% of all patients with the congenital condition DiGeorge syndrome. Symptoms of the disorder can include congenital heart problems, hyperparathryroidism and athymia. Primary immunodeficiency diseases are the result of the athymia, and can lead to the inability to produce normally functioning T cells.

T cells are responsible for defending against infection and regulating essential processes in the immune system. If cDGS goes untreated, it can lead to death within the first 2 years of life.

"We are pleased to welcome Morgan to our growing team," said Alvin Shih, MD, Chief Executive Officer of Enzyvant in a press release. "Morgan's extensive experience in rare disease commercialization will help us achieve our mission of delivering therapeutics to rare disease patients. The Rare Pediatric Disease designation underscores the importance of this mission, and we look forward to continuing to work with the FDA to advance the development of RVT-802."

Rare Pediatric Disease designation is granted by the FDA to therapies with the potential to treat serious or life-threatening rare diseases, specifically conditions that affect individuals under 18 years old. With the designation, pending BLA approval, RVT-802 will be eligible to receive a rare pediatric disease priority review voucher, which can be worth upward of several million dollars.

Earlier this year, Sarepta sold its Priority Review Voucher for $125 million.

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