RARE Daily

FDA Accidentally Publishes Priority Review Voucher Award Ahead of PDUFA Date

October 1, 2021

The U.S. Food and Drug Administration accidentally published a notice in the Federal Register that it was issuing a Priority Review voucher to rare disease drug developer Enzyvant, before approving its treatment.  

The FDA is slated to decide whether or not to approve Enzyvant’s experimental tissue-based regenerative therapy RVT-802 for pediatric congenital athymia on October 8.

The FDA told news sources that the publication was a mistake.

Pediatric congenital athymia is a rare and deadly condition associated with complete DiGeorge Anomaly, CHARGE syndrome, and FOXN1 deficiency. Children with the condition are born without a thymus, resulting in a severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. Approximately 20 infants are born each year in the United States with congenital athymia, which is fatal if untreated. Death typically occurs in the first 24 months of life due to susceptibility to infection. Currently, there are no FDA-approved therapies for this condition.

RVT-802 is derived from infant thymus tissue and stimulates and facilitates the body’s production of naive, immunocompetent T cells, with the goal of bolstering the immune system and restoring the body’s ability to fight infection. Investigational RVT-802 is designed to be administered as a single treatment.

The FDA granted RVT-802 Rare Pediatric Disease designation for DiGeorge Anomaly in September 2017.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 individuals in the U.S. If a Biologics License If the drug is approved, the drugmaker is eligible to receive a priority review voucher, which can be used to reduce the FDA review time on a new drug application to six months from ten months. The vouchers are also potentially lucrative because they are transferrable.

Author: Rare Daily Staff

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