Prevail Therapeutics’ PR001 Receives Orphan Drug Designation and Rare Pediatric Disease Designation from FDA
Prevail Therapeutics Inc. (PRVL)
Last prevail therapeutics inc. earnings: 11/12 09:30 am
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Source: GlobeNewswire
Orphan Drug Designation Granted for Treatment of Gaucher Disease Rare Pediatric Disease Designation Granted for Treatment ofNeuronopathic Gaucher Disease NEW YORK, Feb. 12, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (Nasdaq: PRVL) (“Prevail” or the “Company”), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s investigational gene therapy, PR001, for the treatment of patients with Gaucher disease. The Company also announced that the FDA has granted Rare Pediatric Disease Designation for PR001 for the treatment of neuronopathic Gaucher disease (nGD), the most severe form of the condition. “We are pleased to receive these important designations from the FDA, which underscore the critical nature of our work,” said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executi
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