The U.S. Food and Drug Administration (FDA) has recently granted approval for Ogsiveo (nirogacestat) tablets, marking a significant milestone as the first-ever approved treatment for adult patients with progressing desmoid tumors—a rare subtype of soft tissue sarcomas.
Desmoid tumors, while non-cancerous, can exhibit local aggressiveness, invading surrounding structures and organs, causing pain, mobility issues, and a decline in the patient’s quality of life. While surgical removal has historically been the preferred treatment, the high risk of tumor recurrence or other health challenges post-surgery has prompted the exploration of systemic therapies, targeting the entire body, in clinical trials.Â
Richard Pazdur, M.D., the director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases, expressed the agency’s commitment to addressing unmet medical needs, particularly in the realm of rare tumors. Desmoid tumors, being rare and capable of causing severe pain and disability, lacked approved treatment options beyond surgery and radiation.
The approval of Ogsiveo represents a significant step forward in providing a therapeutic solution for patients facing this challenging condition. The efficacy of Ogsiveo was assessed through an international, multicenter, randomized, double-blind, placebo-controlled trial involving 142 adult patients with progressing desmoid tumors deemed unsuitable for surgery.
Patients were randomly assigned to receive either 150 milligrams of Ogsiveo or a placebo orally, twice daily, until disease progression or unacceptable toxicity. The primary efficacy endpoint was progression-free survival, indicating the duration a person is alive without cancer growth or spread. An additional measure of efficacy was the objective response rate, representing tumor shrinkage.Â
Results from the pivotal clinical trial demonstrated that Ogsiveo brought about clinically meaningful and statistically significant improvements in progression-free survival compared to the placebo. The objective response rate also exhibited a substantial difference between the two groups, with a response rate of 41% in the Ogsiveo arm and 8% in the placebo arm.
These findings were further supported by an assessment of patient-reported pain, favoring the Ogsiveo-treated group. Noteworthy is the fact that Ogsiveo was granted Priority Review by the FDA, a designation that expedites the review process for drugs addressing serious conditions and promising significant improvements over existing therapies.
The drug also received Fast Track and Breakthrough Therapy designations, underscoring its potential in addressing the unmet medical needs associated with desmoid tumors. Additionally, Ogsiveo was granted Orphan-Drug designation, providing incentives to support and promote drug development for rare diseases like desmoid tumors.Â
The most common side effects observed in at least 15% of patients in the clinical trial included diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea. The FDA’s approval of Ogsiveo signifies a breakthrough in the treatment landscape for patients with desmoid tumors, offering a viable option beyond traditional surgical interventions.
The drug’s effectiveness in improving progression-free survival and reducing tumor burden, coupled with its well-tolerated side effect profile, positions it as a promising advancement in the care of individuals facing the challenges of desmoid tumors. The various designations received by Ogsiveo underscore its importance in addressing the unmet needs of patients grappling with this rare and often debilitating condition.Â
News Reference Â
FDA, FDA Approved Ogsiveo for Rare Desmoid Tumors, https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-rare-type-non-cancerous-tumors. Â
