SpringWorks Therapeutics, a Stamford-based clinical-stage biopharmaceutical company focusing on medicines for underserved patient populations, has received fast track designation from the U.S. Food and Drug Administration for nirogacestat. The drug is designed to treat adults with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.
Desmoid tumors are noncancerous growths that occur in the connective tissue and can result in pain, internal bleeding, disfigurement and limited range of motion. According to SpringWorks Therapeutics, between 900 and 1,500 new cases of desmoid tumors are diagnosed each year in this country. The FDA granted orphan drug designation for nirogacestat in June for the treatment of desmoid tumors, and SpringWorks is planning to begin a DeFi Study – a global Phase 3, double-blind, randomized, placebo-controlled trial – during the first half of 2019.
“The fast track designation recognizes that desmoid tumors have a substantial impact on the lives of patients, and that nirogacestat has the potential to address the significant needs faced by this community who currently do not have an FDA-approved treatment,” said Saqib Islam, CEO of SpringWorks Therapeutics. “We look forward to continuing to work closely with the FDA throughout our development program with the hope of ultimately bringing this important medicine to patients.”