The US Food and Drug Administration (FDA) has approved SpringWorks Therapeutics’ Ogsiveo (nirogacestat) as the first treatment for desmoid tumours, a rare type of non-cancerous soft tissue growth.
Ogsiveo has been specifically approved for use in adults with progressing desmoid tumours who require systemic treatment.
Each year, an estimated 1,650 people in the US are diagnosed with desmoid tumours, which may invade surrounding structures and organs, resulting in pain, mobility issues and decreased quality of life.
Although surgical removal has historically been the treatment of choice, desmoid tumours are associated with recurrence rates of up to 77% following surgical resection, and treatment guidelines now recommend systemic therapies as first-line intervention instead of surgery for most tumour locations requiring treatment.
Saqib Islam, chief executive officer of SpringWorks, said: “This community has been waiting for an effective treatment that not only shrinks their tumours but also significantly improves pain, which is the most debilitating symptom reported by people living with desmoid tumours.
“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.”
The FDA’s decision was supported by positive results from the late-stage DeFi trial, in which Ogsiveo was associated with a clinically meaningful and statistically significant improvement in progression-free survival compared to placebo.
The objective response rate was also statistically different between the two arms, with a response rate of 41% in the Ogsiveo cohort and 8% in the placebo.
Mrinal Gounder, an investigator in the DeFi trial, said: “As a treating physician, it was encouraging to see in the DeFi trial that Ogsiveo achieved statistically significant and clinically meaningful improvements across the primary and all key secondary endpoints, while also having a manageable safety profile. This approval represents an important therapeutic advance for patients.”
The FDA’s decision comes just two weeks after SpringWorks announced positive top-line results from a phase 2b study evaluating its investigational MEK inhibitor mirdametinib in a subset of paediatric and adult patients with neurofibromatosis type 1, a rare genetic disorder with an estimated global birth incidence of approximately one in 3,000 individuals.