2023 Highlights
OGSIVEO (nirogacestat) Approved!
The U.S. Food and Drug Administration (FDA) has approved OGSIVEO™ (nirogacestat) for the treatment of adults with progressing desmoid tumors! Below is the DTRF press release with the details: (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 for 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 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 to be successful they needed the support of a patient advocacy organization. A 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.
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