Enzyvant Begins Rolling Submission of BLA for Single Administration cDGA Treatment.
M2 PHARMA-July 13, 2018-Enzyvant Begins Rolling Submission of BLA for Single Administration cDGA Treatment
(C)2018 M2 COMMUNICATIONS
- Basel, Switzerland- and Cambridge, Massachusetts-based biopharmaceutical company Enzyvant has initiated its rolling submission of a biologics license application (BLA) for RVT-802 to the US FDA, the company said.
RVT-802 is an investigational, single administration, tissue-based regenerative therapy designed to treat the primary immune deficiency resulting from congenital athymia associated with complete DiGeorge Anomaly (cDGA) which is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.
As part of the FDA's commitment to expedite therapeutics that aim to address high unmet medical needs, the agency has agreed to a rolling BLA submission process for RVT-802. RVT-802 has been designated as breakthrough therapy, regenerative medicine advanced therapy, rare pediatric disease, and orphan drug by the FDA.
Enzyvant is focused on developing innovative treatments for patients with rare diseases. The company is currently working on RVT-802 for complete DiGeorge Anomaly and RVT-801, an investigational enzyme replacement therapy for the treatment of Farber disease.
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|Date:||Jul 13, 2018|
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