The European Commission (EC) has granted marketing authorisation to SpringWorks Therapeutics, a subsidiary of Merck KGaA, for Ogsiveo (nirogacestat) to treat desmoid tumours – rare and locally aggressive tumours that arise in the body’s connective tissues.
Between 1,300 to 2,300 new cases are diagnosed each year in the European Union.
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The oral gamma secretase inhibitor is approved as a single agent for adults with progressing desmoid tumours requiring systemic treatment.
Ogsiveo approval is supported by findings from the Phase III DeFi trial, which included 142 adult participants with progressing desmoid tumours.
The global, double-blind, randomised and placebo-controlled study assessed the safety, efficacy and tolerability of nirogacestat in adults.
It achieved the primary endpoint by demonstrating an improvement in progression-free survival (PFS).
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By GlobalDataOgsiveo also showed a statistically significant reduction in the risk of disease progression of 71% compared to placebo.
The objective response rate (ORR) was also significantly higher in the Ogsiveo group, with 41% achieving a confirmed response compared to 8% in the placebo group.
Merck Healthcare CEO and executive board member Danny Bar-Zohar stated: “Ogsiveo is already established as the standard of care systemic therapy for desmoid tumours in the US, and our goal is to bring the same treatment benefits to patients in Europe.
“Following last month’s EC approval of our therapy for patients with NF1-PN, we are in the unique position of launching two innovative treatments – underscoring our commitment to the rare tumour patient community.”
Ogsiveo is already approved in the US for the treatment of adults with progressing desmoid tumours.
The US Food and Drug Administration and the European Medicines Agency have both designated Ogsiveo as an orphan drug for this indication.
