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.