Pharma Focus Asia

Prevail Therapeutics Announces FDA Orphan Drug Designation Granted to PR006 for the Treatment of Patients with Frontotemporal Dementia with a GRN Mutation

Tuesday, December 17, 2019

Prevail Therapeutics Inc., 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 (ODD) for the company’s gene therapy candidate, PR006, for the treatment of patients with frontotemporal dementia (FTD) with a GRN mutation (FTD-GRN). PR006 is designed to increase progranulin levels in FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

Orphan Drug designation is granted by the U.S. Food and Drug Administration to drugs or biologics intended to treat a rare disease or condition, which is a disease or condition that affects fewer than 200,000 individuals in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.

“FTD with a GRN mutation is characterized by progressive difficulties in decision-making, behavior and language,” said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Prevail. “With no approved treatments for FTD, there is an urgent unmet need for therapies that slow or stop this disease. Orphan drug designation is an important milestone as we prepare to bring PR006 into the clinic in the first half of 2020.”

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