The Medicines and Healthcare products Regulatory Agency has approved nirogacestat hydrobromide (Ogsiveo) for adults with progressing desmoid tumours, using the International Recognition Procedure with the European Medicines Agency as reference. The MHRA says it will continue close safety monitoring, including advice on contraception and a pregnancy prevention card. ([gov.uk](Link

Desmoid tumours are not malignant but can be invasive, painful and function‑limiting, most often in the abdomen, limbs or trunk. Incidence is typically estimated at 2–4 cases per million annually, with a higher prevalence in women aged 20–44. That rarity often translates into long diagnostic journeys and repeated surgeries before systemic options are considered. ([ncbi.nlm.nih.gov](Link

Nirogacestat is an oral gamma‑secretase inhibitor that dampens NOTCH signalling, aiming to slow or shrink tumours. In the phase 3 DeFi trial (142 adults), nirogacestat reduced the risk of progression or death by around 71% versus placebo (HR 0.29) and achieved a 41% objective response rate versus 8% on placebo; patient‑reported pain and function also improved. ([pmc.ncbi.nlm.nih.gov](Link

Safety will be a key part of roll‑out. The MHRA highlights common side effects such as diarrhoea, rash, nausea, fatigue, hypophosphataemia, headache and stomatitis; ovarian dysfunction/premature menopause can occur and the medicine must not be used in pregnancy. A patient card supports pregnancy prevention and counselling. ([gov.uk](Link

Today’s licence follows the EU marketing authorisation granted on 14 August 2025, with SpringWorks Therapeutics Ireland Limited listed as the EU authorisation holder. The UK decision recognises that EU assessment underpinned the MHRA’s reliance route. ([ema.europa.eu](Link

Access now moves to evaluation by the National Institute for Health and Care Excellence. NICE lists ‘Nirogacestat for treating desmoid tumours’ (ID6453) under a Single Technology Appraisal, with the first committee on 15 April 2026 and final guidance due 10 June 2026, subject to change. ([nice.org.uk](Link

If NICE issues a positive recommendation on 10 June 2026, the NHS statutory funding requirement means routine commissioning should start within 90 calendar days-by 8 September 2026-unless NICE specifies otherwise (for example, phased adoption). Local formularies must then reflect the guidance. ([nice.org.uk](Link

Pricing will be closely watched. A UK list price has not yet been published. For context only, U.S. company commentary at launch referenced a list price of about $29,000 for a 30‑day supply; UK net costs will depend on confidential commercial terms and any patient access scheme agreed through the NICE process. ([managedhealthcareexecutive.com](Link

Budget impact should be manageable relative to large oncology launches given the small eligible population, but final uptake will depend on NICE’s cost‑effectiveness verdict and any commercial arrangement. From April 2026, NICE’s updated value‑for‑money thresholds take effect, which could support access where evidence of benefit is strong. ([nice.org.uk](Link

Manufacturing and supply sit within a changing corporate backdrop: SpringWorks Therapeutics was acquired by Merck KGaA, Darmstadt, Germany in 2025, positioning Ogsiveo within a larger rare‑tumour portfolio for Europe, including the UK. ([emdgroup.com](Link

For clinicians and patients, the near‑term focus is straightforward: confirm eligibility against the licensed indication, discuss benefits and risks-especially fertility and pregnancy considerations-and report adverse events via the Yellow Card scheme while real‑world outcomes accumulate. ([gov.uk](Link

For investors and SME leaders, the milestones are clear: NICE committee on 15 April, potential final guidance on 10 June, and-if positive-NHS commissioning within the 90‑day window. Watch for the publication of the UK product information (SmPC/PIL) and any commercial agreement that shapes the budget impact for 2026/27. ([nice.org.uk](Link