Will This Dementia Treatment Be a Big Hit After FDA Decision?
Prevail Therapeutics Inc. (NASDAQ: PRVL) shares jumped early on Tuesday after the firm announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) for its dementia treatment.
Specifically, the designation is for the firm’s gene therapy candidate, PR006, for the treatment of patients with frontotemporal dementia (FTD) with a GRN mutation. There are currently no approved therapies for FTD.
ODD is granted by the FDA 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 ODD status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.
Asa Abeliovich, M.D., Ph.D., founder and CEO of Prevail, commented:
FTD with a GRN mutation is characterized by progressive difficulties in decision-making, behavior and language. 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.
Shares of Prevail Therapeutics closed Monday at $15.33, in a 52-week range of $7.41 to $16.90. The consensus price target is $18.33. Following the announcement, the stock was up over 11% at $17.10 in early trading indications Tuesday.