FDA Approves First Therapy for Progressing Desmoid Tumors

On Monday, November 27, 2023, the United States Food and Drug Administration (FDA) approved Ogsiveo (nirogacestat), making it the first FDA-approved treatment for desmoid tumors. The drug is intended for adult patients with desmoid tumors that need systemic therapies.

Ogsiveo is an oral small-molecule gamma-secretase inhibitor manufactured by SpringWorks Therapeutics.

The approval was based on an international, multicenter, randomized, double-blind, placebo-controlled clinical trial. Throughout the trial, researchers recruited over 140 patients with desmoid tumors who were not good candidates for surgical resection.

Patients were divided into experimental and control groups receiving 150 mg of oral Ogsiveo or a placebo twice daily.

The researchers determined that Ogsiveo patients had significantly higher progression-free survival than the placebo group. According to the study results published in the New England Journal of Medicine, Ogsiveo reduced the risk of disease progression by 71%. Additionally, the objective response rate, measured by tumor shrinkage, was 41% in the experimental group, compared to 8% in the control group.

Despite the efficacy demonstrated by the drug, the study also identified potential risks. Approximately 15% of patients had side effects, including diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, respiratory tract infections, and dyspnea.

“This approval is the culmination of a collaborative effort between the patient community, academia, and the biopharmaceutical industry, who worked together with tenacity and persistence to advance promising science,” said Jeanne Whiting, Executive Director Emeritus and Co-Founder of the Desmoid Tumor Research Foundation, in the by SpringWorks Therapeutics press release.

“Our hope is that patients and their families will benefit from greater awareness of desmoid tumors, faster diagnoses, and better outcomes now that there is an approved and effective treatment.”  

According to the FDA statement, desmoid tumors are a rare non-cancerous soft tissue sarcoma that can be locally aggressive. The administration reveals that these tumors can encroach on surrounding structures and organs, causing pain, mobility issues, and reduced quality of life. Before a therapeutic alternative became available, desmoid tumors were primarily treated through surgical resection. However, the risk of tumor recurrence or surgical complications prompted research on systemic treatments.

The availability of alternative treatment options provides additional tools for providers and more choices for patients.

“The FDA continues to address unmet medical need and advance the development of safe and effective therapies for the millions of Americans whose lives are affected by rare tumors,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in the FDA statement.