A breakthrough for people with a rare type of soft tissue tumour has been hailed following the approval of a new drug in the USA.
Biopharmaceutical company SpringWorks Therapeutics has announced that the US Food and Drug Administration (FDA) has approved OGSIVEO™ (nirogacestat) for the treatment of adults with progressing desmoid tumours. This is the first FDA-approved medical therapy in this rare disease.
This means nirogacestat will be made available for patients in the USA. The FDA approval is based on the results of a phase 3 trial, which showed that the drug reduced the risk of disease progression and improved other symptoms such as pain.
Patients in the UK won’t be able to get access to nirogacestat yet. SpringWorks expects to file a Marketing Authorisation Application for OGSIVEO to the European Medicines Agency in the first half of 2024. If this decision is positive, it would then be assessed by NICE to decide whether it should be made available on the NHS.
Desmoids are classed as intermediate tumours that sit between non-cancerous and cancerous tumours. This means that they have the capacity to come back at or near the original tumour, but they do not spread. They are sometimes referred to as desmoid tumours or aggressive fibromatosis. Patients are treated by sarcoma specialist oncologists who have the skills and experience to treat them. In every million people, about 5-6 could be expected to develop a desmoid tumour.
Sarcoma UK’s Director of Research, Policy and Support, Dr Sorrel Bickley, said: “This is hopeful news for people with desmoid tumours. Living with a desmoid can be painful and debilitating, so having an effective drug could make a big difference. We hope OGSIVEO will also be available to patients in the UK soon and we look forward to representing patient views as part of the regulatory approvals process.”