RARE Daily

SpringWorks Gets Breakthrough Therapy Designation for Desmoid Tumor Drug

August 30, 2019

FDA Grants SpringWorks Breakthrough Therapy Designation for Desmoid Tumor Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has granted SpringWorks Therapeutics Breakthrough Therapy designation for nirogacestat, its experimental drug for the treatment of adult patients with the rare and progressive cancer known as desmoid tumors.

Nirogacestat is an oral, selective, small molecule gamma-secretase inhibitor in phase 3 clinical development for the treatment of desmoid tumors. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which is believed to play a role in activating pathways that contribute to desmoid tumor growth.

Desmoid tumors are rare and often debilitating and disfiguring soft-tissue tumors that can aggressively invade surrounding healthy tissues and cause significant morbidities, including severe pain, internal bleeding, incapacitating loss of range of motion, and, in rare cases, death. It is estimated that 1,000 to 1,500 new desmoid tumor patients are diagnosed each year in the United States. There are currently no therapies approved by the FDA for the treatment of desmoid tumors.

“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,” said Saqib Islam, CEO of SpringWorks. “We are currently enrolling adult patients in our phase 3 DeFi trial and will continue to work closely with the FDA with the goal of bringing nirogacestat to patients as quickly as possible.”

The FDA’s Breakthrough Therapy designation is designed to expedite the development and regulatory review of medicines that are intended to treat a serious condition. To qualify for this designation, preliminary clinical evidence must demonstrate that the medicine may provide substantial improvement over currently available therapy on at least one clinically significant endpoint.

Previously, the FDA had granted nirogacestat Orphan Drug designation for the treatment of desmoid tumors and Fast Track designation for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.

Photo: Saqib Islam, CEO of SpringWorks

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