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The Desmoid Tumor Research Foundation

Breaking News: Springworks Therapeutics Announces Submission Of New Drug Application For Nirogacestat To The U.S. FDA

Pharmaceutical News

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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. 

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*Update: Watch the 2022 DTRF Annual Patient Meeting presentations on the Nirogacestat results and the AL102 results. For all Patient Meeting recordings, visit Patient Meeting.*

BIG NEWS this Desmoid Tumor Awareness Month!

Presentations on the latest data from two promising clinical trials – both studying gamma secretase inhibitor drugs in desmoid tumors – were given this past weekend at the 2022 European Society for Medical Oncology (ESMO) conference in Paris, France.

PLEASE NOTE: Presentations tailored for the patient community on these two trials and their data will be given at the Annual Patient Meeting Webinar #2 (of 3) on Sept. 24th during the DTRF Together We Will Virtual Weekend. Register at no cost at DTRF.org/weekend, and bring your questions!

Both of these presentations have left us encouraged and hopeful. The interest in desmoid tumor research is higher than ever before and we are making significant progress in finding more effective and more manageable therapies. If you have fundraised for or donated to the DTRF, you have helped make this progress possible by supporting our patient outreach, advocacy, research, awareness, and scientific collaboration efforts. Give today.

Here are the details from the ESMO presentations:

  • NIROGACESTAT – Phase 3 DeFi Clinical Trial (SPRINGWORKS THERAPEUTICS)

On Saturday, Principal Investigator Bernd Kasper, MD, (University of Heidelberg, Maneheim) presented the latest data from the SpringWorks Therapeutics Phase 3 DeFi clinical trial for nirogacestat (a gamma secretase inhibitor) in desmoid tumor patients during the Presidential Symposium session of ESMO.

“Results showed a statistically significant improvement in progression-free survival in patients randomised to nirogacestat compared to the placebo group, with a 71% lower risk of disease progression on average. The response rate was also much higher – 41% in the nirogacestat arm and only 8% in the placebo arm; nearly one in ten patients (7%) showed a complete response with the agent.” 

– Bernd Kasper, MD, PhD, University of Heidelberg, Principal Investigator of the DeFi trial

For more information, see the SpringWorks’ press release

…and the ESMO article.

  • AL102 – RINGSIDE Phase 2/3 Clinical Trial (AYALA PHARMACEUTICALS)

On Monday, Prof. Robin Jones, MD, (The Royal Marsden) presented the latest data from the Ayala Pharmaceuticals RINGSIDE Phase 2/3 clinical trial for AL102 (a gamma secretase inhibitor) in desmoid tumor patients.

“Efficacy was demonstrated across all cohorts of Part A of the study, with early responses that deepened over time. The results also showed that AL102 was well-tolerated across all doses.” 

– Roni Mamluk, PhD, CEO of Ayala


For more information, read Ayala’s press release

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On May 24, 2022, SpringWorks Therapeutics announced topline results from their Phase 3, double-blind, placebo-controlled trial called the DeFi trial. This trial evaluates the gamma secretase inhibitor, nirogacestat, for efficacy, safety and tolerability in adult patients with progressing desmoid tumors.

The DeFi trial’s primary endpoint was met by improving progression-free survival (PFS) for usage of nirogacestat compared to placebo, with a 71% reduction in the risk of desmoid tumor progression.

SpringWorks Therapeutics expects to present additional data at an upcoming medical conference as well as submit a New Drug Application (NDA) to the U.S. Food and Drug Administration in the second half of 2022.

From SpringWorks Therapeutics’ press release:

“‘Desmoid tumors are aggressive soft-tissue tumors that can lead to severe negative outcomes for patients, including long-lasting pain, disfigurement, and amputation. In rare cases, when vital organs are impacted, desmoid tumors can also be life-threatening,’ said Saqib Islam, Chief Executive Officer of SpringWorks. ‘Today’s announcement represents a significant milestone towards our goal of bringing the first approved therapy to the desmoid tumor community. We look forward to sharing the DeFi trial data with the FDA and to presenting detailed study results at a medical meeting later this year.’”

The DTRF would like to express our gratitude to members of the community who have participated in and are currently enrolled in clinical trials, including the DeFi trial. Your participation is advancing our understanding of desmoid tumors and bringing us closer to improved treatments for all desmoid tumor patients.