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Pharmaceuticals / Submissions and Approvals

Enzyvant Gains Two FDA Therapy Designations for its Drug Candidate RVT-802

April 24, 2017
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Enzyvant was granted an FDA regenerative medicine advanced therapy designation and a breakthrough therapy designation for its investigational therapy RVT-802.

The therapy is being developed for complete DiGeorge syndrome, which is characterized by congenital heart disease, hypoparathyroidism and athymia in pediatric patients.

Preliminary clinical results suggest a survival rate of over 70 percent for treated patients, according to the company.

In addition to these designations, RVT-802 has been granted orphan drug designation by the FDA. Enzyvant anticipates a BLA filing in the first half of 2018.

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