Springworks’ nirogacestat receives EC orphan status

pharmafile | September 26, 2019 | News story | Research and Development  

Springworks, a clinical-stage biopharmaceutical company focusing on severe rare disease and cancer has been granted orphan status by the European Commission for its oral, small molecule, selective gamma-secretase inhibitor nirogacestat in the treatment of soft tissue sarcoma.

The drug company is currently enrolling patients in phase III trials assessing nirogacestat for the treatment of desmoid tumours, which are a type of soft-tissue tumours that are often treated by sarcoma specialists.

Saqib Islam, Springworks’ Chief Executive 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 nirogecstat 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.”

Desmoid tumours are rare and often debilitating and disfiguring, and can aggressively invade surrounding healthy tissues and cause significant morbidities including severe pain, internal bleeding, incapacitating loss of range of motion and, in rare cases, death.

In the United States alone it is estimated that 1,000 to 1,500 new desmoid tumour patients are diagnosed each year for which there are no therapies approved by the US FDA.

Nirogacestat previously received Orphan Drug Status from the FDA in June 2018.

Nik Kiran

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