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First-Ever Drug Approval for Rare Soft Tissue Sarcomas
The U.S. Food and Drug Administration (FDA) has given the green light to Ogsiveo (nirogacestat) tablets for adult patients grappling with progressing desmoid tumors necessitating systemic treatment. This landmark decision marks the inaugural approval of a drug specifically tailored for patients combating desmoid tumors, a rare subtype of soft tissue sarcomas.
Desmoid tumors, though non-cancerous, exhibit local aggressiveness. They possess the potential to invade adjacent structures and organs, inflicting pain, mobility issues, and a diminished quality of life. While surgical removal has traditionally been the preferred treatment, the high recurrence risk or ensuing health complications post-removal have driven the exploration of systemic therapies through clinical trials.
Dr. Richard Pazdur, Director of the FDA’s Oncology Center of Excellence, remarked, “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.”
The efficacy of Ogsiveo was evaluated in a comprehensive international trial involving 142 adult patients with progressing desmoid tumors ineligible for surgery. Patients were randomly assigned to receive 150 milligrams of Ogsiveo or a placebo orally, twice daily until disease progression or unacceptable side effects surfaced. The trial’s primary efficacy measure was progression-free survival, complemented by an assessment of tumor shrinkage or objective response rate.
Significantly, the pivotal trial demonstrated that Ogsiveo substantially improved progression-free survival compared to the placebo. Moreover, the objective response rate was markedly higher in the Ogsiveo group, registering a response rate of 41% compared to 8% in the placebo group. These findings were reinforced by patient-reported pain assessments, favoring the Ogsiveo-treated cohort.
The most prevalent side effects observed in at least 15% of the patients included diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.
Ogsiveo’s approval pathway was expedited, having received Priority Review, Fast Track, Breakthrough Therapy, and Orphan Drug designations. The latter specifically supports drug development for rare diseases, providing incentives to encourage advancement in treatment.
The FDA granted approval for Ogsiveo to SpringWorks Therapeutics Inc., signaling a promising stride in addressing unmet medical needs for individuals battling desmoid tumors.
