The Medicines and Healthcare products Regulatory Agency (MHRA) announced today, January 7, 2026, the approval of Nirogacestat hydrobromide, marketed as Ogsiveo, for the treatment of progressing desmoid tumours in adults. This decision marks a significant advancement in the management of these rare, non-cancerous tumours.
Desmoid tumours, although benign, can cause substantial damage as they grow within connective tissues, often affecting the arms, legs, or abdomen. The newly approved drug, Nirogacestat hydrobromide, functions by inhibiting specific proteins that contribute to tumour growth. Clinical studies have demonstrated that patients treated with Nirogacestat experience prolonged periods without disease progression, potentially reducing the need for surgical intervention.
Understanding Desmoid Tumours
Desmoid tumours, also known as aggressive fibromatosis, are rare and complex. While they do not metastasize like cancer, their aggressive nature can lead to significant morbidity. The tumours are notorious for their unpredictable growth patterns and can be challenging to manage due to their tendency to recur after surgical removal.
According to the Desmoid Tumour Research Foundation, these tumours affect approximately 1 to 2 individuals per million annually. The approval of Nirogacestat offers hope to those whose lives are impacted by this condition, providing a non-surgical option that targets the underlying mechanisms of tumour growth.
Expert Opinions and Regulatory Insights
Julian Beach, the MHRA Interim Executive Director for Healthcare Quality and Access, emphasized the importance of this approval. “Patient safety is our top priority,” he stated. “The approval of Nirogacestat hydrobromide will benefit adults with desmoid tumours, improving health and quality of life. As with all licensed medicines, we will continue to monitor its safety and effectiveness closely.”
“The approval of Nirogacestat hydrobromide will benefit adults with desmoid tumours, improving health and quality of life.” – Julian Beach, MHRA
The MHRA’s decision aligns with a growing trend towards personalized medicine, where treatments are tailored to specific biological pathways. This approach not only enhances efficacy but also minimizes unnecessary side effects, a critical consideration for chronic conditions like desmoid tumours.
Potential Side Effects and Safety Measures
While Nirogacestat offers promising benefits, it is not without potential side effects. Common reactions include diarrhoea, rash, nausea, fatigue, hypophosphataemia, headache, and stomatitis. More serious concerns, such as premature menopause, may affect more than 1 in 10 individuals.
Particularly noteworthy is the drug’s potential impact on fertility. Nirogacestat may harm an unborn baby if taken during pregnancy and could affect ovarian and testicular function. Consequently, it is imperative that women of childbearing potential use highly effective contraception while undergoing treatment. A patient card will be provided to support pregnancy prevention, containing crucial information on risk mitigation strategies.
A comprehensive list of side effects will be available in the Patient Information Leaflet (PIL) and the Summary of Product Characteristics (SmPC), to be published on the MHRA website within a week of approval.
Reporting and Monitoring
Patients and healthcare providers are encouraged to report any adverse effects through the MHRA’s Yellow Card scheme, accessible online or via mobile applications. This feedback mechanism is vital for ongoing safety monitoring and helps ensure that the benefits of Nirogacestat continue to outweigh any risks.
The approval of Nirogacestat hydrobromide represents a significant milestone in the treatment of desmoid tumours, offering a new therapeutic option that could transform the standard of care for affected patients. As the medical community continues to explore the full potential of this drug, ongoing research and patient monitoring will be crucial in optimizing its use and ensuring it meets the highest safety standards.
