MHRA Approves Nirogacestat Hydrobromide for Desmoid Tumour Treatment
The Medicines and Healthcare products Regulatory Agency (MHRA) announced the approval of Nirogacestat hydrobromide (Ogsiveo) on January 7, 2026. This medicine is indicated for the treatment of progressing desmoid tumours in adult patients.
Understanding Desmoid Tumours
Desmoid tumours are non-cancerous growths that develop in connective tissue, commonly found in the arms, legs, or abdomen. While not malignant, their growth can result in damage to adjacent tissues and may present challenges in surgical removal.
Mechanism of Action and Efficacy
Nirogacestat hydrobromide functions by inhibiting specific proteins implicated in the proliferation of these tumours. Clinical studies have indicated that patients receiving nirogacestat hydrobromide experienced a longer period without disease progression and a reduced requirement for surgical intervention.
Safety Information and Side Effects
Commonly reported side effects associated with nirogacestat hydrobromide include:
- Diarrhoea
- Rash
- Nausea
- Tiredness
- Hypophosphataemia (low blood phosphate levels)
- Headache
- Stomatitis (mouth lining inflammation)
A serious side effect, premature menopause, may affect over 10% of individuals.
Nirogacestat hydrobromide carries a risk of harm to an unborn baby if administered during pregnancy. The impact on ovarian and testicular function, and consequently on male and female fertility, remains under investigation. Therefore, the medication is contraindicated during pregnancy, and highly effective contraception must be employed by patients.
A patient information card will be distributed to support pregnancy prevention measures for female patients using nirogacestat hydrobromide and for female partners of male patients on the drug. This card provides crucial information regarding potential risks and necessary mitigation steps.
Comprehensive details on all side effects will be available in the Patient Information Leaflet (PIL) and Summary of Product Characteristics (SmPC), which will be published on the MHRA website within seven days of the approval date. Individuals experiencing suspected side effects are advised to consult their doctor, pharmacist, or nurse and report the effects via the Yellow Card scheme.