Approval Granted for Nirogacestat Hydrobromide to Treat Desmoid Tumours
The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Nirogacestat hydrobromide (Ogsiveo) for treating progressing desmoid tumours in adults as of 7 January 2026. This decision is significant as it offers a new treatment option for a rare but debilitating condition.
Understanding Desmoid Tumours
Desmoid tumours develop in the body’s connective tissues, primarily affecting the arms, legs, and abdomen. Although these tumours are not cancerous, their growth can lead to damage of nearby tissues, making surgical removal challenging.
How Nirogacestat Works
Nirogacestat hydrobromide functions by blocking specific proteins that contribute to tumour growth. Clinical studies indicate that patients using this medication have experienced extended periods without disease progression, potentially reducing the need for surgical interventions.
Statement from the MHRA
Julian Beach, Interim Executive Director for Healthcare Quality and Access at MHRA, said:
Ensuring patient safety remains our foremost concern. The endorsement of nirogacestat hydrobromide promises to enhance the health and quality of life for adults suffering from desmoid tumours.
We will proceed to monitor the safety and effectiveness of the medicine rigorously.
Side Effects and Precautions
Common adverse effects of nirogacestat hydrobromide include diarrhoea, rash, nausea, fatigue, low phosphate levels, headaches, and mouth inflammation. A notable risk involves premature menopause, which may occur in more than 10% of users.
Additionally, there are concerns regarding its impact during pregnancy, as the drug may harm an unborn baby. Due to uncertainties surrounding its effects on fertility, highly effective contraception is advised for both male and female patients during treatment.
A patient card will be issued to assist in pregnancy prevention for both female patients and partners of male patients using this medication. This card will contain vital information regarding the associated risks and necessary precautions.
Resources and Reporting Side Effects
A comprehensive list of side effects is available in the Patient Information Leaflet (PIL) and the Summary of Product Characteristics (SmPC), which are set to be published on the MHRA website within a week of approval. Those who suspect they are experiencing side effects should consult their healthcare provider and can report issues via the Yellow Card scheme, accessible through its website or mobile app.
Background
The approval of Nirogacestat hydrobromide follows the International Recognition Procedure (IRP) and was based on assessments by the European Medicines Agency. The MHRA oversees the regulation of medical products in the UK, ensuring both efficacy and safety for patients.
Source: official statements, news agencies, and public reports.
https://www.gov.uk/government/news/nirogacestat-hydrobromide-approved-to-treat-desmoid-tumours
