US-based SpringWorks Therapeutics has received orphan drug designation from the European Commission (EC) for its small molecule candidate nirogacestat to treat soft tissue sarcoma.
EC grants orphan drug status to products designed to treat or prevent life-threatening or severe conditions affecting less than five individuals out of 10,000 in the European Union.
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Nirogacestat is an oral, selective inhibitor of gamma-secretase. It splits various transmembrane protein complexes, including Notch, which is involved in the activation of pathways related to the growth of desmoid tumours.
Desmoid tumours are rare soft tissue tumours. Their growth pattern invades surrounding healthy tissues such as joints, muscle and viscera.
These types of tumours cause significant morbidities, including internal bleeding, severe pain, incapacitating loss of range of motion and even death, in rarer cases. Typical treatment includes surgical resection, which is, however, associated with a high recurrence rate.
Previously, nirogacestat secured orphan drug, fast track and breakthrough therapy designations from the US regulatory agency in progressive, unresectable, recurrent or refractory desmoid tumours or deep fibromatosis indication.
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By GlobalDataApproximately 1,000 to 1,500 individuals are diagnosed with desmoid tumours each year in the US. The tumours currently lack approved therapies.
Nirogacestat is now undergoing Phase III development for desmoid tumours.
SpringWorks Therapeutics CEO Saqib Islam said: “This orphan drug designation in the European Union is another important development for SpringWorks and follows the orphan drug, fast track and breakthrough therapy designations already granted for nirogacestat in the US by the FDA.
“We are currently enrolling adult patients in our DeFi trial and will continue to work closely with global regulators with the goal of bringing nirogacestat to patients as quickly as possible.”
DeFi is a Phase III clinical trial assessing the drug candidate in adults with progressing desmoid tumours.
