The Desmoid Tumor Research Foundation

Update: Springworks Therapeutics Has Announced FDA acceptance And Priority Review Of The New Drug Application For Nirogacestat

FDA

SpringWorks Therapeutics has announced FDA acceptance and priority review of the New Drug Application (NDA) for nirogacestat, their investigational gamma secretase inhibitor for the treatment of adults with desmoid tumors.

“The acceptance of our NDA for nirogacestat with Priority Review represents a significant milestone in our ambition to provide the first approved therapy for patients with desmoid tumors. We look forward to working closely with the FDA during the review process and remain focused on ensuring that we are well-positioned to expeditiously serve the desmoid tumor patient and the physician communities following approval.”

Saqib Islam, CEO, SpringWorks Therapeutics

Read the press release

What This Means

This announcement brings our community one step closer to an FDA-approved therapy for desmoid tumors. The FDA now has until August 27, 2023 to complete the review process, which will lead to a decision of whether nirogacestat will receive FDA approval. This is very exciting!

Related News

The DeFi phase 3 trial results that lead to this NDA were published in The New England Journal of Medicine. The publication and NDA are remarkable accomplishments that demonstrate the power of collaboration between patient groups, researchers and industry.

Read DTRF Press Release.
Read “Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors” on NEJM.org.

SpringWorks Therapeutics has announced that it has submitted a New Drug Application to the U.S. FDA for nirogacestat, their investigational gamma secretase inhibitor for the treatment of adults with desmoid tumors. 

“Our NDA submission represents an important step toward potentially delivering the first approved therapy for patients with desmoid tumors.” 

– Saqib Islam, CEO, SpringWorks Therapeutics 

Read the press release

What Happens Next 

Once submitted, the FDA has about 60 days to decide whether to “accept” the New Drug Application (NDA). If accepted, the official FDA review process will start. 

A standard FDA review process is typically about 10 months. SpringWorks has applied for various regulatory designations that the FDA has in place to review drugs that treat serious conditions and fulfill an unmet need. The FDA granted nirogacestat Fast-Track and Breakthrough Therapy Designations in the past, and SpringWorks also plans to submit the NDA for review under the FDA’s Real-Time Oncology Review program. 

All of this is good news and means that there is recognition that there is an important need for a new drug like nirogacestat.

Because there are many variables with the FDA approval process, SpringWorks is only able to say right now that it expects the FDA to make a decision sometime in 2023. As more precise timelines become available, SpringWorks will let the DTRF know, and we will then be able to update our community.

To learn more about the nirogacestat Phase 3 clinical trial results, please watch the recording of Dr. Ravin Ratan’s (MD Anderson Cancer Center) presentation from the 2022 DTRF Annual Patient Meeting in September.