MHRA Approves Nirogacestat Hydrobromide for Progressing Desmoid Tumours
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A new treatment option is now available for adults in the UK battling progressing desmoid tumours. The Medicines and Healthcare products Regulatory Agency (MHRA) approved nirogacestat hydrobromide (marketed as Ogsiveo) on January 7, 2026, offering hope for improved health and quality of life for those affected by this challenging condition.
Understanding Desmoid Tumours
Desmoid tumours are growths that develop in the body’s connective tissues, most commonly appearing in the arms, legs, or abdomen. While not cancerous, these tumours can be locally aggressive, causing significant damage to surrounding tissues and presenting difficulties in surgical removal. The approval of Nirogacestat hydrobromide addresses a critical unmet need for patients facing this complex illness.
How Nirogacestat Hydrobromide Works
Nirogacestat hydrobromide functions by inhibiting the activity of specific proteins crucial to the growth of desmoid tumours. Clinical studies have demonstrated that patients receiving the medication experienced a longer period without disease progression and, importantly, a reduced need for surgical intervention.
According to a senior official at the MHRA, “Patient safety is our top priority. The approval of nirogacestat hydrobromide will benefit adults with desmoid tumours, improving health and quality of life.” The agency emphasized its commitment to ongoing monitoring of the drug’s safety and effectiveness post-approval.
Potential Side Effects and Safety Considerations
As with all medications, nirogacestat hydrobromide carries potential side effects. The most commonly reported include diarrhoea, rash, nausea, tiredness, hypophosphataemia (low phosphate levels), headache, and stomatitis (inflammation of the mouth). A more serious side effect, affecting more than 1 in 10 people, is premature menopause.
Crucially, the medication poses a significant risk to developing babies and may impact fertility in both men and women. Therefore, nirogacestat hydrobromide is strictly contraindicated during pregnancy, and patients – both female and male – must utilize highly effective contraception while undergoing treatment. To support this, a dedicated patient card outlining the risks and necessary precautions will be provided to female patients and the female partners of male patients.
A comprehensive list of side effects will be available in the Patient Information Leaflet (PIL) and Summary of Product Characteristics (SmPC), to be published on the MHRA website within seven days of approval.
Reporting Adverse Reactions
Individuals experiencing suspected side effects from nirogacestat hydrobromide are strongly encouraged to consult with their doctor, pharmacist, or nurse. Side effects can also be reported directly to the MHRA’s Yellow Card scheme via their website (https://yellowcard.mhra.gov.uk/) or through the MHRA Yellow Card mobile app, available on the Google Play and Apple App stores.
Regulatory Pathway and Further Information
The approval was granted on January 7, 2026, through the International Recognition Procedure (IRP), with the European Medicines Agency (EMA) serving as the Reference Regulator (EMEA/H/C/006071/0000). Detailed information, including the Summary of Product Characteristics and Patient Information Leaflets, will be accessible on the MHRA Products website within seven days.
The MHRA plays a vital role in ensuring the safety and efficacy of all medicines and medical devices in the UK, operating as an executive agency of the Department of Health and Social Care. Media inquiries can be directed to [email protected] or by calling 020 3080 7651.
