Tuesday, November 28, 2023
Today, the U.S. Food and Drug Administration (FDA) has granted approval for Ogsiveo (nirogacestat) tablets, marking a significant milestone as the first drug sanctioned for the treatment of adult patients dealing with advancing desmoid tumors—a rare subtype of soft tissue sarcomas that necessitate systemic intervention.
Desmoid tumors, though non-cancerous, exhibit local aggressiveness, infiltrating neighboring structures and organs, leading to pain, mobility issues, and diminished quality of life. While surgical removal has conventionally been the preferred treatment, the associated high risk of tumor recurrence or other health complications post-surgery has prompted increased exploration of systemic therapies in clinical trials.
Dr. Richard Pazdur, Director of the FDA's Oncology Center of Excellence and Acting Director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research, emphasized the FDA's commitment to addressing unmet medical needs, particularly for rare tumors affecting millions of Americans. He noted, "Desmoid tumors are rare tumors that can lead to severe pain and disability. Today's approval will offer the first approved treatment option for patients beyond surgery and radiation."
Ogsiveo's efficacy was assessed in a global, multicenter, randomized, double-blind, placebo-controlled trial involving 142 adult patients with progressing desmoid tumors not suitable for surgery. Patients were randomly assigned to receive 150 milligrams (mg) of Ogsiveo or placebo orally twice daily until disease progression or intolerable toxicity. Progression-free survival (the duration a person is alive without cancer growth or spread) was the primary efficacy measure, with objective response rate (indicating tumor shrinkage) as an additional outcome.
The pivotal trial demonstrated that Ogsiveo yielded clinically meaningful and statistically significant improvements in progression-free survival compared to the placebo. The objective response rate also significantly differed between the two groups, with a response rate of 41% in the Ogsiveo group compared to 8% in the placebo group. These findings were reinforced by patient-reported pain assessments favoring the Ogsiveo group.
Common side effects observed in at least 15% of the trial participants included diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.
Ogsiveo was granted Priority Review, Fast Track, and Breakthrough Therapy designations by the FDA, reflecting the drug's potential to significantly enhance the safety and efficacy of treating desmoid tumors. Additionally, it received Orphan-Drug designation, providing incentives for the development of drugs targeting rare diseases. SpringWorks Therapeutics Inc. was granted FDA approval for Ogsiveo.
Source: prnewswire.com
