FDA Grants Enzyvant Rare Pediatric Disease Designation for DiGeorge Syndrome Cell Therapy

September 5, 2017

Rare Daily Staff

The U.S. Food and Drug Administration granted Enzyvant Rare Pediatric Disease Designation for RVT-802, an experimental tissue-based therapy for primary immune deficiency associated with the rare and fatal condition Complete DiGeorge Syndrome.

The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18. If Enzyvant is successful in winning marketing approval for RVT-802, it will receive a rare pediatric disease priority review voucher.

The voucher is potentially lucrative because it is transferrable and can be used to accelerate the approval of any drug. That could give the holder faster access to mutibillion markets. In the past, such vouchers have sold for as much as $350 million.

The FDA previously granted Enzyvant Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, and Orphan Drug designation for RVT-802.

About 1 percent of people with the congenital condition DiGeorge Syndrome have Complete DiGeorge Syndrome. This is characterized by congenital heart disease, low calcium levels caused by hypoparathyroidism, and a severe immunodeficiency caused by the lack of thymus tissue. The lack of thymus tissue, known as athymia, leaves people unable to produce normally functioning T cells, the workhorses of the immune system that fight infection and regulate immune system processes.

RVT-802 is an experimental cell-based therapy that uses proprietary processes to harvest, culture, and apply thymic tissue from other people to treat primary immune deficiency associated with Complete DiGeorge Syndrome.

Enzyvant said it expects to file RVT-802 for FDA marketing approval in the first half of 2018.

September 5, 2017

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