The Medicines and Healthcare products Regulatory Agency (MHRA) has today (7\u00a0January\u00a02026) approved the medicine\u00a0Nirogacestat\u00a0hydrobromide (Ogsiveo)\u00a0for the treatment of\u00a0progressing desmoid tumours in adults.\u00a0<\/p>\n
Desmoid tumours are tumours that form in\u00a0connective tissue in the body, usually the arms, legs or abdomen.\u00a0Whilst they are\u00a0not cancerous,\u00a0as\u00a0they\u00a0grow,\u00a0they\u00a0can\u00a0cause damage in nearby tissue\u00a0and become difficult to remove.\u00a0\u00a0<\/p>\n
Nirogacestat\u00a0hydrobromide\u00a0works by inhibiting\u00a0the activity of certain proteins\u00a0which are involved in the growth of the tumours.\u00a0Studies have shown that patients taking\u00a0nirogacestat\u00a0hydrobromide\u00a0benefitted from\u00a0living longer\u00a0without their condition getting worse\u00a0and avoiding the need for potential surgery.\u00a0\u00a0<\/p>\n
Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said:<\/strong>\u00a0\u00a0<\/p>\n Patient safety\u00a0is our top priority.\u00a0\u00a0<\/p>\n The approval of\u00a0nirogacestat\u00a0hydrobromide\u00a0will\u00a0benefit\u00a0adults with desmoid tumours, improving\u00a0health and quality of life.\u00a0\u00a0<\/p>\n As with all licensed medicines, we will continue to monitor its safety and effectiveness closely.<\/p>\n<\/blockquote>\n The most common side effects\u00a0of taking\u00a0nirogacestat hydrobromide include diarrhoea, rash, nausea (feeling sick), tiredness, hypophosphataemia (low blood levels of phosphates), headache,\u00a0and\u00a0stomatitis (inflammation of the lining of the mouth).\u00a0A serious side effect can be premature menopause, which may affect more than 1 in 10 people.\u00a0<\/p>\n Nirogacestat\u00a0hydrobromide may cause harm to an unborn baby if taken during pregnancy. It is uncertain how it may damage ovaries and testicles, and\u00a0how it may affect men and women\u2019s fertility. Therefore,\u00a0nirogacestat\u00a0hydrobromide must not be taken during pregnancy, and highly effective contraception must be used.\u00a0<\/p>\n A patient card will be provided to support pregnancy prevention in female patients taking\u00a0nirogagestat\u00a0hydrobromide, and in female partners of male patients taking this drug. The card contains important information on the risks and the measures that must be followed to minimise potential harm.\u00a0<\/p>\n A full list of side effects can be found in the Patient Information Leaflet (PIL) or the Summary of Product Characteristics (SmPC), which will be published on the MHRA website within 7 days of approval.\u00a0<\/p>\n\n