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FDA Greenlights First Treatment for Uncommon Non-Cancerous Growth | FDA Approvals | iPharmaCenter

The U.S. Food and Drug Administration sanctioned Ogsiveo (nirogacestat) tablets for adult patients grappling with advancing desmoid tumours necessitating systemic intervention. Ogsiveo marks a groundbreaking approval as the inaugural drug greenlit for addressing patients with desmoid tumours, an unusual subtype within the spectrum of soft tissue sarcomas.

Desmoid tumours, though non-malignant, exhibit local aggressiveness. These growths may encroach upon neighbouring structures and organs, resulting in discomfort, compromised mobility, and diminished quality of life. Historically, surgical excision has been the favoured recourse; however, the recurrence risk or post-removal complications have prompted an increasing exploration of systemic therapies (comprehensive body-targeted cancer treatments) in clinical trials.

Ogsiveo's efficacy underwent scrutiny in a global, multi-centre, randomized, double-blind, placebo-controlled trial involving 142 adult patients with advancing desmoid tumours unsuitable for surgery. Participants were randomly assigned to orally receive 150 milligrams (mg) of Ogsiveo or a placebo twice daily until disease progression or intolerable adverse effects. The primary efficacy benchmark was progression-free survival (the duration a person remains alive without cancer progression or spread post-treatment initiation). An ancillary efficacy metric was the objective response rate (indicating tumour reduction).

The pivotal clinical trial unveiled Ogsiveo's provision of clinically significant and statistically notable enhancements in progression-free survival compared to the placebo. Furthermore, the objective response rate showed statistical disparities between the arms, registering a 41% response rate in the Ogsiveo group versus 8% in the placebo group. The progression-free survival outcomes found support in patient-reported pain assessments that favoured the Ogsiveo arm.

Common adverse effects noted in over 15% of trial participants encompassed diarrhoea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnea.

Ogsiveo earned Priority Review, entailing the FDA's commitment to rendering a decision on an application within six months if the drug, upon approval, would substantially enhance safety or effectiveness in treating, diagnosing, or preventing a severe condition compared to existing therapies. Ogsiveo also secured FDA Fast Track and Breakthrough Therapy designations for the specified indication, alongside Orphan-Drug designation for desmoid tumour (aggressive fibromatosis) treatment. Orphan-drug designation furnishes incentives to foster and support drug development for rare diseases.

The FDA bestowed approval for Ogsiveo upon SpringWorks Therapeutics.

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