SpringWorks Therapeutics Gains FDA Endorsement for Tumor Drug

Author: Paul Schott, Stamford Advocate
Published On: 09/30/2019

STAMFORD — Biotech firm SpringWorks Therapeutics has gained an endorsement from the U.S. Food and Drug Administration to help expedite the review of a drug that would treat rare tumors.

The “Breakthrough Therapy Designation” supports the development of Nirogacestat, an oral medicine for desmoid tumors, which are rare soft-tissue tumors that can cause pain, internal bleeding and disfigurement and limit patients’ physical motion. Desmoid tumors do not yet have any FDA-authorized therapies, and the agency would still need to make a final approval before the drug could go on the market.

“We are committed to pursuing the rapid development of Nirogacestat given the important need for new therapies for patients with desmoid tumors and are pleased to receive this Breakthrough Therapy Designation,” SpringWorks CEO Saqib Islam said in a statement. “We are currently enrolling adult patients in our phase-three ‘DeFi’ trial and will continue to work closely with the FDA with the goal of bringing Nirogacestat to patients as quickly as possible.”

With pharmaceutical giant GlaxoSmithKline, SpringWorks is also developing Nirogacestat to treat multiple myeloma, a rare cancer. GSK contributed an undisclosed amount to SpringWorks’ recent “Series B” funding round.

The FDA would separately review Nirogacestat’s treatments for multiple myeloma and desmoid tumors.

Among other recent milestones, SpringWorks announced in June that it had secured an FDA “fast track” designation that will accelerate the agency’s review of “PD-0325901,” an oral therapy for patients 2 years old and older who have certain forms of Neurofibromatosis Type 1, a genetic disorder caused by mutations in the NF1 gene.

Also in June, SpringWorks announced a partnership with Chinese biotech firm BeiGene. The alliance will focus on developing treatments for mutations identified in solid tumors that drive cancer growth.

Spun off from Pfizer in 2017, SpringWorks relocated last November to 100 Washington Blvd. in Stamford’s South End. It occupies about 24,000 square feet of space in the waterfront office tower.

“There’s clearly a revitalization effort going on in Stamford,” Islam said in an interview earlier this year. “Most of what’s attracted us to it is this area’s connectivity north and south, both getting to Manhattan and Boston, which are two major poles of influence in pharma and biotech.”

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