Merck’s therapy becomes first authorised option for aggressive soft tissue tumours
Merck has announced that the European Commission has granted marketing authorisation for Ogsiveo (nirogacestat) as monotherapy for adults with progressing desmoid tumors requiring systemic treatment.
Ogsiveo is now the first and only approved therapy for desmoid tumors in the European Union.
Bernd Kasper, Professor at Mannheim Cancer Center and principal investigator of the phase 3 DeFi trial, said: “Ogsiveo is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumor symptoms, including a significant reduction in pain.”
Lynne Hernandez, Executive Director of the Desmoid Tumor Research Foundation, added: “This approval is a long-awaited advance for desmoid tumor patients, their families and physicians in Europe.”
Desmoid tumors are rare, locally aggressive soft tissue growths that affect around 1,300 to 2,300 people annually in the EU. They can cause severe pain, disfigurement and loss of mobility, and are known for their high recurrence rates.
The EC approval is based on data from the phase 3 DeFi trial, which enrolled 142 adults. Ogsiveo showed a 71% reduction in risk of disease progression compared to placebo and a confirmed objective response rate of 41% versus 8%.
Patients receiving Ogsiveo also reported early and sustained improvements in pain, physical functioning and overall quality of life.
The most common adverse reactions included diarrhoea, rash, ovarian toxicity, nausea, fatigue and headache.
Ogsiveo is already approved in the US and has received Orphan Drug designation from both the FDA and EMA. Merck says the launch of Ogsiveo, alongside its recent approval for NF1-PN, underscores its commitment to rare tumour communities.