US FDA approves SpringWorks Ogsiveo to treat adults with desmoid tumours

SpringWorks Therapeutics, Inc., a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced that the US Food and Drug Administration (FDA) has approved Ogsiveo (nirogacestat), an oral gamma secretase inhibitor, for the treatment of adult patients with progressing desmoid tumours who require systemic treatment. The FDA previously granted breakthrough therapy, fast track and orphan drug designations to nirogacestat for the treatment of desmoid tumours. 

“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 tumours are locally aggressive and invasive soft-tissue tumours that can lead to substantial morbidity. In addition, when vital structures are impacted, desmoid tumours can be life-threatening. Although they do not metastasize, desmoid tumours are often refractory to existing off-label systemic therapies and associated with recurrence rates of up to 77% following surgical resection. Desmoid tumour experts and treatment guidelines now recommend systemic therapies as first-line intervention instead of surgery for most tumour locations requiring treatment.

“Desmoid tumours can have a significant impact on people’s lives and are difficult to manage due to their invasive nature and high rates of recurrence. Ogsiveo is a highly innovative therapy with efficacy data demonstrating both meaningful antitumor activity and a significant improvement in desmoid tumour symptoms,” said Mrinal M. Gounder, M.D., sarcoma medical oncologist at Memorial Sloan Kettering Cancer Center (MSK) in New York City and an investigator in the phase 3 DeFi trial. “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 approval of Ogsiveo is based on the results from the phase 3 DeFi trial, which were published in the March 9, 2023 edition of the New England Journal of Medicine. Ogsiveo met the primary endpoint of improving progression-free survival (PFS), demonstrating a statistically significant improvement over placebo with a 71% reduction in the risk of disease progression (hazard ratio (HR) = 0.29 (95% CI: 0.15, 0.55); p< 0.001). Median PFS was not reached in the Ogsiveo arm and was 15.1 months in the placebo arm. Confirmed objective response rate (ORR) based on RECIST v1.1 was 41% with Ogsiveo versus 8% with placebo (p<0.001); the complete response rate was 7% in the Ogsiveo arm and 0% in the placebo arm. The median time to first response was 5.6 months with Ogsiveo and 11.1 months with placebo. PFS and ORR improvements were in favour of Ogsiveo regardless of baseline characteristics including sex, tumour location, tumour focality, treatment status, previous treatments, mutational status, and history of familial adenomatous polyposis. Ogsiveo also demonstrated early and sustained improvements in patient-reported outcomes (PROs), including pain (p<0.001), desmoid tumour-specific symptoms (p<0.001), physical/role functioning (p<0.001), and overall health-related quality of life (p=0.01). 

Ogsiveo exhibited a manageable safety and tolerability profile. The most common adverse events (>15%) reported in patients receiving Ogsiveo were diarrhoea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection, and dyspnoea.

“Today is an extraordinary day for the desmoid tumour community. This approval is the culmination of a collaborative effort between the patient community, academia and the biopharmaceutical industry, who worked together with tenacity and persistence to advance promising science,” said Jeanne Whiting, executive director Emeritus and co-founder of the Desmoid Tumor Research Foundation. “Our hope is that patients and their families will benefit from greater awareness of desmoid tumours, faster diagnoses, and better outcomes now that there is an approved and effective treatment.” 

SpringWorks is dedicated to helping patients with desmoid tumours access OGSIVEO and to providing support throughout their treatment journey. As part of this commitment, the company is introducing SpringWorks CareConnections, a comprehensive patient support programme that offers personalized services to eligible Ogsiveo patients, including insurance coverage information and access support, financial assistance and personalized educational and emotional support.

Ogsiveo will be available to order through a specialty pharmacy and specialty distributor network in the United States within five to ten business days. SpringWorks expects to file a Marketing Authorisation Application for Ogsiveo in desmoid tumours with the European Medicines Agency in the first half of 2024.

Ogsiveo (nirogacestat) is an oral, selective, small molecule gamma secretase inhibitor approved in the United States for the treatment of adult patients with progressing desmoid tumours who require systemic treatment.

Ogsiveo is not approved for the treatment of any other indication in the United States, or for any indication in any other jurisdiction by any other health authority.

SpringWorks is also evaluating nirogacestat as a potential treatment for patients with ovarian granulosa cell tumours and for patients with multiple myeloma as part of several B-cell maturation agent (BCMA) combination therapy regimens in collaboration with leaders in industry and academia.

SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer.

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