Merck KGaA’s recently acquired SpringWorks unit has claimed EU approval for Ogsiveo, becoming the first approved therapy for desmoid tumours in Europe.
Oral gamma secretase inhibitor Ogsiveo (nirogacestat) has been cleared by the European Commission as a monotherapy for adults with progressing desmoid tumours who require systemic treatment.
It is the second major approval for Merck since it closed the $3.9 billion takeover of US biotech SpringWorks, coming after the EU approval for oral MEK inhibitor Ezmekly (mirdametinib) – used to treat tumours in people with neurofibromatosis type 1 – last month.
Both medicines have already been approved in other markets, including the US, and are a key component of Merck’s bid to build a presence in rare tumours.
The EU approval comes on the back of the phase 3 DeFi trial, which enrolled 142 adult patients with progressing desmoid tumours, a form of benign but locally aggressive growth that affects soft tissues. They do not spread to other parts of the body like cancer, but can be debilitating, and – in rare cases, when vital structures are impacted – even life-threatening.
Traditionally, treatment has concentrated on surgery, radiotherapy, and/or chemotherapy, but systemic treatment is now generally recommended as the frontline option, if possible, as recurrence rates after surgery can be as high as 77%.
In DeFi, treatment with Ogsiveo achieved a 71% reduction in disease progression compared to placebo, with an objective response rate (ORR) of 41% – including 7% complete responses – versus 8% in the control group.
“Desmoid tumours can have a profound impact on people’s lives and are difficult to manage due to their invasive nature and high rates of recurrence,” said Bernd Kasper, of the University of Heidelberg, principal investigator of the DeFi trial.
“Until now, there have been no approved medicines in Europe,” he added. “Ogsiveo is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumour symptoms, including a significant reduction in pain, which is the most debilitating symptom reported by patients.”
Approximately 1,300 to 2,300 new cases of desmoid tumours are diagnosed annually in the EU, according to SpringWorks, which now operates as a wholly owned subsidiary of Merck.
The German group acknowledged at an R&D update in that year that it had a pressing need to bolster its pipeline, particularly through external ‘bolt-on’ partnership deals, and get drugs through development and onto the market more quickly. It has set a target of launching a new product every 18 months.
