SpringWorks's Ogsiveo Bags USFDA Nods to Treat Desmoid Tumours

SpringWorks Therapeutics, Inc., a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced that Ogsiveo (nirogacestat), an oral gamma secretase inhibitor, has been approved by the US Food and Drug Administration (FDA) for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. 

The FDA previously granted breakthrough therapy, fast track and orphan drug designations to nirogacestat for the treatment of desmoid tumours, as per pharmabiz.  

“Our team is honoured to deliver the first FDA-approved therapy for patients with desmoid tumours. This community has been waiting for an effective treatment that not only shrinks their tumors but also significantly improves pain, which is the most debilitating symptom reported by people living with desmoid tumours,” said Saqib Islam, chief executive officer of SpringWorks. "We are pleased with the broad label, which includes all progressing adult patients and specifically references improvement in pain, and believe Ogsiveo has the potential to become the new standard of care for people living with these devastating tumours. This is a watershed moment for the desmoid tumour community and we would like to extend our gratitude to the patients, families, investigators, and advocacy groups involved in the journey to making Ogsiveo available in the US.”

Desmoid tumors are aggressive and invasive soft-tissue tumors that can cause significant morbidity. Furthermore, when critical structures are harmed, desmoid tumors can be fatal. Although they do not spread, desmoid tumors are frequently resistant to existing off-label systemic medications and have recurrence rates of up to 77% after surgical excision. 

Systemic treatments, rather than surgery, are currently recommended as first-line therapy for most desmoid tumor sites by specialists and treatment recommendations.