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The Desmoid Tumor Research Foundation

Update: OGSIVEO® (nirogacestat) Now Available in 150 mg and 100 mg Tablets

SpringWorks

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In recent months, the DTRF learned from patients that the 50mg tablet of Ogsiveo® (nirogacestat) will no longer be available after 2024. The DTRF contacted SpringWorks Therapeutics to advocate for patients who are currently receiving the 50mg tablet.

Last week, the SpringWorks Therapeutics team confirmed that the 50mg tablet of Ogsiveo® will, in fact, no longer be available as of January 1, 2025.  

New blister packs are available for the FDA-approved 100mg and 150mg doses of Ogsiveo®. Click here to read the statement SpringWorks provided to the DTRF.

The DTRF advises that patients talk to their physician, pharmacist, or other trusted medical team member with any questions. Medical professionals are encouraged to reach out to the SpringWorks Medical Information team on behalf of their patients to receive answers to questions regarding this change.

Follow the steps below with the help of your medical team to receive the most timely and accurate Ogsiveo® information: 

  1. Bring your Ogsiveo® dosage questions or concerns directly to a trusted member of your medical team (including your physicians and pharmacists).
  2. Ask your trusted medical professional to contact a SpringWorks Medical Information team member directly at 1-888-400-7989 or medinfo@springworkstx.com.
  3. Request that your medical professional ask specific, direct questions when discussing Ogsiveo® so that clear recommendations can be relayed back to their patient(s).

Please note: Only medical professionals, including physicians, pharmacists, or other medical team members, are permitted to discuss Ogsiveo® dosage questions or concerns with the SpringWorks Medical Information team.  

If you are a desmoid tumor patient needing educational, emotional, or financial support and resources while taking Ogsiveo®, you can connect directly with SpringWorks CareConnections™. 

If you’ve been impacted by the 50mg tablet no longer being available, please contact the DTRF at info@dtrf.org to share your experience and help us continue advocating for desmoid tumor patients.

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Thank you to our global desmoid tumor community of patients, caregivers, researchers, and medical professionals who traveled to Houston and participated in the DTRF Together We Will Weekend. It was wonderful to connect with everyone in person in a brand new city! We are grateful to our platinum event sponsor, SpringWorks Therapeutics, for making the Weekend possible.

Together we advanced research, connected with each other, empowered ourselves, and fundraised for a cure, while celebrating our community and Desmoid Tumor Awareness Month!

  • September 20, 2024 – DTRF International Desmoid Tumor Research Workshop: Dedicated clinicians and researchers from around the world attended the Int’l Desmoid Tumor Research Workshop, presenting the latest desmoid tumor science and research advances. We’re honored to host this essential collaborative meeting each year, which helps steer the direction of desmoid tumor research and the Foundation’s funding priorities. Learn more about the annual DTRF International Desmoid Tumor Research Workshop.
  • September 21, 2024 – DTRF Patient Meeting: At the heart of our annual Weekend, patients, caregivers, clinicians, and researchers gathered to connect, collaborate and learn. Presentations on research, treatments, and a special presentation on the patient experience. Recordings of these educational events (including live Q&A) are available on our Patient Meeting Presentations page.
  • September 22, 2024 – 2024 RFA Walk: Our community gathered together in person at the Houston Zoo and fundraised virtually around the world. Together, more than $55,000 was raised for desmoid tumor research! 

The work continues to find better treatments and, one day, a cure for each and every desmoid tumor patient. Once again, on behalf of the DTRF team, THANK YOU!

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(Pittsburgh, PA) – November 27, 2023 – The U.S. Food and Drug Administration (FDA) announced approval today of OGSIVEO (nirogacestat) for the treatment of adults with progressing desmoid tumors. This is the first FDA-approved medical therapy in this rare disease. FDA approval follows positive results from the SpringWorks Therapeutics, Inc. (Nasdaq: SWTX) DeFi (NCT03785964) phase 3 clinical trial, a global, randomized, double-blind, placebo-controlled trial evaluating nirogacestat in adult patients with desmoid tumors. Results from the DeFi trial were published on March 9, 2023, in The New England Journal of Medicine in the article, “Nirogacesat, a γ-Secretase Inhibitor for Desmoid Tumors,” by Drs. Gounder, Ratan, Kummar, Kasper et al.

OGSIVEO (nirogacestat) is an oral gamma secretase inhibitor. For more information, visit www.ogsiveo.com.

“The significance of FDA approval of OGSIVEO (nirogacestat) to desmoid tumor patients cannot be overstated. This is an extraordinary victory in the area of rare diseases, and demonstrates the power of collaboration among patients, industry and researchers.”

Jeanne Whiting, Executive Director Emeritus, Desmoid Tumor Research Foundation

“SpringWorks is grateful to the Desmoid Tumor Research Foundation, as well as the patients and investigators who participated in our trials, whose enduring commitment and enthusiasm helped us get to this point. We are honored to provide the first FDA-approved therapy and committed to offering ongoing support to the desmoid tumor community throughout their treatment journey on OGSIVEO.”

Saqib Islam, Chief Executive Officer, SpringWorks Therapeutics

The collaborative path to approval

The Desmoid Tumor Research Foundation (DTRF) has been instrumental in the nearly 15-year path to approval of OGSIVEO (nirogacestat). The drug was first studied in patients with a range of solid tumors including seven desmoid tumor patients. A poster presentation of the promising results of this early work in desmoid tumors caught the eye of members of the National Cancer Institute’s (NCI) Rare Tumors Initiative, Shivaani Kummar, MD and Abby Sandler, PhD. The pair were interested in a phase 2 trial, but due to the rare nature of the disease, concluded that in order to be successful they needed the support of a patient advocacy organization. Synergistic collaboration between the DTRF and NCI brought the phase 2 trial forward, and with the DTRF’s outreach efforts, filled it in less than five months. 

Despite promising results that were coming out of the phase 2 study, a business decision resulted in halting the development of the asset. Determined not to let this potential treatment stall, the DTRF once again galvanized its resources and continued discussions with Pfizer staff. Supportive clinicians, as well as encouraging staff at Pfizer, supported the effort to move the drug forward. Eventually SpringWorks Therapeutics was created to license nirogacestat and other assets that showed potential in underserved patient populations.

Under newly-formed SpringWorks, the phase 3 DeFi trial for nirogacestat opened. During the conduct of the DeFi trial, and with the help of DTRF’s patient outreach, the trial was fully enrolled in July 2020. In September 2022, trial data were presented in a Presidential Symposium during the ESMO Congress and at the annual DTRF International Desmoid Tumor Research Workshop. In December 2022, SpringWorks filed a New Drug Application for nirogacestat which was accepted for Priority Review on February 27, 2023. Today, FDA approval of OGSIVEO (nirogacestat), makes it the first FDA-approved drug for the treatment of adults with desmoid tumors.

“This breakthrough represents the power of patient advocacy. The Desmoid Tumor Research Foundation’s role in OGSIVEO’s path to approval represents collaboration at its finest. Were it not for their advocacy work to shepherd the drug through the trial phases, facilitate communication among stakeholders, and use their outreach to help fill the trial, we may not be here today. I’m honored to be part of the process that brings the first-ever FDA-approved medical therapy to desmoid tumor patients.”

Mrinal Gounder, MD, Memorial Sloan Kettering Cancer Center

About desmoid tumors

Desmoid tumors (also known as aggressive fibromatosis) are rare, invasive, soft tissue tumors that form in the connective tissues of the body. They affect 5-6 per 1 million people per year. Although they do not metastasize, desmoid tumors can be locally aggressive and severely damage surrounding tissues and vital structures as they grow. They are highly recurrent and can be multifocal. These tumors may occur anywhere in the body (abdominal, intraabdominal, and extra-abdominal locations) and approximately 5-10% arise in the context of a rare genetic condition known as Familial Adenomatous Polyposis (F.A.P.). Previously, there have been no approved therapies for desmoid tumors.

About The Desmoid Tumor Research Foundation

The Desmoid Tumor Research Foundation (DTRF) was co-founded in 2005 by Jeanne Whiting and Marlene Portnoy, with the mission of facilitating and funding desmoid tumor research and supporting patients with information. The DTRF’s mission is to aggressively fund research to accelerate the development of improved therapies, and ultimately find a cure for desmoid tumors. The DTRF collaborates with dedicated researchers and clinicians worldwide to facilitate research and improve the lives of patients through education, awareness and support. Every year the foundation’s progress has gained momentum with new clinical trials, emerging therapies and important new discoveries about the disease coming to fruition. The DTRF is a recipient of the National Organization of Rare Disorders Abby S. Meyers Leadership Rare Impact Award, and the Global Genes’ Champions of Hope Rare Champion in Advocacy Award. For more information, please visit www.DTRF.org.

For more information about this important milestone for desmoid tumor patients, read the FDA’s and SpringWorks’ press releases linked below.

SpringWorks Therapeutics Press Release

U.S. FDA Press Release

DTRF Press Release PDF

Media Inquiries

Maggie Cameron
Communications Manager, DTRF
Maggie@DTRF.org

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THANK YOU to our global desmoid tumor community of patients, care partners, researchers, and medical professionals, who traveled to Philadelphia and participated in the DTRF Together We Will Weekend. It was wonderful to be in-person again! We are grateful to our platinum event sponsor, SpringWorks Therapeutics, for making the Weekend possible!

Together we advanced research, connected, empowered ourselves, and fundraised for a cure, while celebrating our community and Desmoid Tumor Awareness Month!

  • September 22, 2023 – DTRF International Desmoid Tumor Research Workshop: Dedicated clinicians and researchers from around the world attended the 10th Annual DTRF International Desmoid Tumor Research Workshop, presenting on the latest desmoid tumor science and research advances. We’re honored to host this important collaborative meeting each year, which helps steer the direction of desmoid tumor research and the Foundation’s funding priorities. Learn more about the annual DTRF International Desmoid Tumor Research Workshop.
  • September 23, 2023 – DTRF Patient Meeting: At the heard of our Weekend, patients, caregivers, clinicians and researchers gathered to connect, collaborate and learn together. Presentations on research, treatments, and a special presentation on the patient experience. Recordings of these educational events (including live Q&A) are available on our Patient Meeting Presentation/Media page.
  • 2023 RFA Virtual Champions Awards Ceremony: Scheduled for Sunday, September 24, 2023, the RFA 5K & Fun Run was cancelled due to a named storm on the East Coast, and we had to pivot to 100% virtual fundraising. Our community did not disappoint. Together, more than $63,000 was raised for desmoid tumor research! On November 2nd, the ceremony was streamed LIVE on Facebook! | WATCH

The work continues to find better treatments and, one day, a cure for each and every desmoid tumor patient! Once again, on behalf of the DTRF team, THANK YOU!

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The Desmoid Tumor Research Foundation Announces That Groundbreaking Results From The Phase 3 Springworks Defi Trial Were Published Today In The New England Journal Of Medicine In The Article, “Nirogacestat, A Γ-Secretase Inhibitor For Desmoid Tumors.” The Fda Recently Accepted The New Drug Application (Nda) For Nirogacestat And It Is Now Under Priority Review.

(Pittsburgh, PA) – Thursday, March 9th, 2023 – Years of advocacy by The Desmoid Tumor Research Foundation (DTRF) facilitated clinical trials in nirogacestat in desmoid tumors. On February 27, 2023, SpringWorks Therapeutics announced FDA acceptance and Priority Review of their New Drug Application (NDA) for nirogacestat, for the treatment of adults with desmoid tumors. The NDA is based on the previously announced positive results from the DeFi (NCT03785964) phase 3 trial, a global, randomized, double-blind, placebo-controlled trial evaluating nirogacestat in adult patients with desmoid tumors. Results from the DeFi trial were published today in The New England Journal of Medicine in the article, “Nirogacesat, a γ-Secretase Inhibitor for Desmoid Tumors,” by Drs. Gounder, Ratan, Kummar, Kasper et al.

This exciting news follows a 14 year, arduous path. Nirogacestat, a γ-secretase inhibitor, was first studied in patients with a range of solid tumors including seven desmoid tumor patients. A poster presentation of the promising results of this work in desmoid tumors caught the eye of a member of the National Cancer Institute’s (NCI) Rare Tumors Initiative. The NCI team, Shivaani Kummar, MD and Abby Sandler, PhD, wanted to run a phase 2 trial, but due to the rare nature of the disease, concluded that they couldn’t be successful without the support of a patient advocacy organization. After they found the DTRF, NCI and the DTRF enthusiastically collaborated to bring the phase 2 trial forward. While it had been anticipated that it might take up to two years to fill this trial, with the DTRF’s outreach to patients, the trial was filled in less than 5 months.

Despite promising results that were coming out of the phase 2 study, a business decision resulted in halting the development of this molecule. Once again, the DTRF galvanized its resources and continued discussions with Pfizer staff, determined not to let this potential treatment stall because it showed such promise for desmoid tumor patients who have limited treatment options and no approved therapies. Supportive clinicians, as well as encouraging staff at Pfizer, supported the effort to move the drug forward. Eventually SpringWorks Therapeutics was created to acquire nirogacestat and other assets that showed potential in underserved patient populations. 

Under newly-formed SpringWorks, the phase 3 DeFi trial for nirogacestat eventually opened. Over the following 15 months, with the help of the DTRF’s outreach, the trial was fully enrolled. In September 2022, final trial data were presented in a Presidential Symposium during the ESMO Congress and at the DTRF’s annual International Desmoid Tumor Research Workshop. In December 2022, SpringWorks filed a New Drug Application for nirogacestat in desmoid tumors which was accepted for Priority Review on February 27, 2023. If approved, nirogacestat could be available to patients as the first FDA-approved therapy in desmoid tumors.

With the support of the DTRF, three additional clinical trials on desmoid tumors are currently underway. For a rare disease with an incidence rate of 5-6 per million, these are remarkable accomplishments that demonstrate the power of collaboration between patient groups, researchers and industry.

About The Desmoid Tumor Research Foundation 

The Desmoid Tumor Research Foundation (DTRF) was co-founded in 2005 by Jeanne Whiting and Marlene Portnoy, with the mission of facilitating and funding desmoid tumor research and supporting patients with information. The DTRF’s mission is to aggressively fund research to accelerate the development of improved therapies, and ultimately find a cure for desmoid tumors. The DTRF collaborates with dedicated researchers and clinicians worldwide to facilitate research and improve the lives of patients through education, awareness and support. Every year the Foundation’s progress has gained momentum with new clinical trials, emerging therapies and important new discoveries about the disease coming to fruition. The DTRF is the recipient of the National Organization of Rare Disorders Abby S. Meyers Leadership Rare Impact Award, and the Global Genes’ Champions of Hope Rare Champion in Advocacy Award. For more information, please visit www.dtrf.org.

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Press Contact:
Maggie Cameron
Communications Manager, DTRF
Maggie@DTRF.org

Download PDF

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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.