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The Desmoid Tumor Research Foundation

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

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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.

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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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Desmoid tumors were in the spotlight at the 2022 Connective Tissue Oncology Society (CTOS) annual conference in Vancouver, Canada last weekend!

Designated the “Sarcoma of the Year” for this year’s event, “Desmoid Fibromatosis” was the focus of an informative special session on Saturday which featured presentations on active surveillance, cryoablation, metronomic therapy, and the DeFi Phase 3 clinical trial results.

The session was chaired by Torsten Nielsen, MD, PhD, (Department of Pathology at Vancouver General Hospital) with moderators Chiara Colombo, N/A, MD (Fondazione IRCCS Istituto Nazionale dei Tumori) and DTRF Medical Advisory board member Bernd Kasper, MD PhD (University of Heidelberg, Mannheim University Medical Center).

We are grateful to CTOS for giving desmoid tumors this highlight, and to the doctors and researchers involved in the hopeful research presented to the global sarcoma medical community.

For more information, watch the recordings of the 2022 DTRF Patient Meeting Webinars on two of the topics mentioned at CTOS:

DTRF Patient Meeting Webinar #2: Cryoablation
DTRF Patient Meeting Webinar #2: Nirogacestat – DeFi Phase 3 Results

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*Update: For recordings and up-to-date CME accredited course information, visit our Get CME Credits page.*

We are pleased to share that PeerView Institute in partnership with the DTRF will provide a unique MasterClass & Practicum educational symposium on desmoid tumors for members of the medical community to increase knowledge of desmoid tumors and treatment among medical professionals.

On November 17th, during the Connective Tissue Oncology Society (CTOS) Annual Meeting, the symposium entitled “On the Cusp of Change: Modernizing the Treatment of Desmoid Tumors with Novel Gamma Secretase Inhibitors” will be presented by Bernd Kasper, MD, PhD (University of Heidelberg), Mrinal Gounder, MD (Memorial Sloan Kettering Cancer Center), and Alessandro Gronchi, MD (Fondazione IRCCS Istituto Nazionale dei Tumori). The faculty will provide expert insights and practical guidance for navigating the latest treatment advances, including gamma-secretase inhibitors (GSIs), for desmoid tumors. Illustrative cases and clinical scenarios will be discussed to demonstrate the emerging role and integration of novel GSIs in real-world patient care settings.

The completion of this symposium will award participants Continuing Medical Education (CME) credits. CMEs are authorized by the American Medical Association Physician’s Recognition Award and are often required to maintain medical licenses and credentials to see patients, providing a strong incentive for physicians to maintain or increase their knowledge and skills over time. 

Physicians can register at https://bit.ly/DesmoidTumors2022T. 

We look forward to this educational event, and thank CTOS, PeerView, and our community for making this important project possible.

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Thank you to the Connective Tissue Oncology Society (CTOS) for hosting “Desmoid Fibromatosis”, a webinar aired in advance of the 2022 CTOS Annual Meeting to further educate sarcoma professionals on #desmoid tumors and the patient experience.

Watch the recording of the event

The DTRF’s Executive Director Jeanne Whiting presented “Patients’ Perspective: Navigating the Desmoid Tumor Journey”, and other presenters covered pathobiology, pathology, and localized and systemic treatment options currently available for desmoid tumors. The webinar ended with a thoughtful dialogue between experts on current challenges.

We are grateful for this coveted spotlight and want to thank all who attended hoping to learn more about this #raredisease and its impacts. 

DTRF Executive Director Jeanne Whiting thanked the community after her presentation:

“I want to thank everyone who helped me share the desmoid tumor patient story in this talk. I received almost a hundred patient comments and stories submitted by email. I wish I could have shared everyone’s story, but I hope I was able to convey the major issues we face along with the emotion and meaning behind your comments. It was a wonderful opportunity to have this special CTOS webinar focused on our disease and to let the patients’ voices be heard!”

We look forward to hearing more about the “Sarcoma of the Year” at #CTOS2022 in November!

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On the heels of another successful Desmoid Tumor Awareness Month, we are thrilled to formally announce The Desmoid Tumor Research Foundation Grant Awards for 2022! 

The proposals from these institutions offer the potential for significant advancements in desmoid tumor research and align with the research priorities set by our Scientific Advisory Board. For more information, visit DTRF-Awarded Grants.

Funding for these critical projects would not be possible without the continued support of our community and donors – YOU! We would like to extend an additional special thank you to the families who have helped fund some of these important projects. 

The awards below are contingent upon a mutually agreed upon grant agreement.

To mark this exciting announcement, please consider sharing this news and/or making a donation today. 

  • Sameer Rastogi, MD, Aiims- All India Institute of Medical Science,New Delhi, “To see the early effect of sorafenib on cognition, cardiovascular and reproductive functions in patients with fibromatosis.”
  • Cristabelle De Souza, PhD, Stanford University, “Activation of c-JUN modulates the transcriptional activity of CD63 serving as a novel target for the treatment of Desmoid Type Fibromatosis.” Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Joanna Przybyl, PhD, Research Institute – Centre universitaire de santé McGill – McGill University Health Centre, “Targeting hexosamine biosynthesis pathway for the treatment of desmoid tumors.” Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Jonathan Noujaim, MD, #CanSaRCC, “Tyrosine Kinase Inhibitors in Desmoid-type Fibromatosis- A Canadian Multicenter Retrospective CanSaRCC Study.” Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Kris Vleminckx, PhD, Universiteit Gent, “Investigating EZH2 as a druggable mediator of immune cell exclusion in desmoid tumors.” Year 2 of 2. Jointly funded by the DTRF and Desmoid Tumour Foundation of Canada (DFC)
  • Gerlinde Wernig, MD, Stanford Medicine, Department of Pathology, “Studying CD47 blockade as an immunotherapy for desmoid-type fibromatosis.” Year 2 of 2.

Thank you for your support in our mission to find a cure for desmoid tumors!