Nirogacestat
Nirogacestat is the first approved medicine for desmoid tumours, locally invasive growths that do not spread but can be crippling; it works by blocking Notch signalling.
DeFi: PFS HR 0.29 vs placebo; ORR 41% vs 8%; pain and physical function improved. Approved US 27 November 2023 (first systemic therapy for desmoid tumours); EU 2025. Ovarian toxicity (75% of women of childbearing potential, largely reversible) is the notable adverse effect. SpringWorks/Merck KGaA.
1.Nirogacestat binds the gamma-secretase complex
- Route
- Oral
- Schedule
- 150 mg twice daily continuously
- Monitoring
- Diarrhoea, ovarian function/hormones, phosphate, liver enzymes, skin, non-melanoma skin cancer
- Medicare
- Part D (self-administered)
Oral, self-administered, so it is a Part D drug: covered through a stand-alone Part D plan or Medicare Advantage drug benefit, usually on the specialty tier with 25 to 33% coinsurance until the annual cap ($2,000 in 2025, $2,100 in 2026).
- Commercial insurance
- covered with prior authorisation
Covered for FDA-labelled and NCCN-listed uses, but almost always behind prior authorisation confirming diagnosis, biomarker and line of therapy; dispensed through a specialty pharmacy. Progressing desmoid tumour requiring systemic therapy.
- Assistance programmes
- SpringWorks CareConnections
- PAN Foundation — Disease-specific co-pay and premium funds; open and closed funds change monthly.
- HealthWell Foundation
- CancerCare Co-Payment Assistance Foundation
- Patient Advocate Foundation Co-Pay Relief
Part D out-of-pocket capped at $2,000 (2025) / $2,100 (2026). Medicare patients cannot use manufacturer co-pay cards; charity funds (PAN, HealthWell, CancerCare) and the Extra Help subsidy are the routes.
Sources: Medicare.gov: Drug coverage (Part D) · Medicare.gov: Costs for Medicare drug coverage (annual out-of-pocket cap). Not medical or financial advice; verify with your plan.
- Notes
- EU-licensed 2025 for desmoid tumours; UK licence and NICE position not yet researched.
- NHS England
- Not routinely funded
Sources: NICE search: nirogacestat. Funding decisions are indication-specific and change monthly; verify with NICE and your treating team.
Compare all products across the US, EU, UK, Japan, China and Australia →
- 27 Nov 2023ApprovalUS
First systemic therapy for desmoid tumours (DeFi) source
- 2025ApprovalEU
European Commission approval source
Approvals
| Region | Year | Indication |
|---|---|---|
| US | 2023 | Progressing desmoid tumours requiring systemic treatment |
| EU | 2025 | Progressing desmoid tumours |
| Adverse event | Any grade | Grade 3+ |
|---|---|---|
| Diarrhoea DeFi | 84% | 16% |
| Ovarian toxicity women of childbearing potential; mostly resolved on stopping | 75% | — |
| Rash | 68% | — |
| Hypophosphataemia | 65% | — |
Events listed without rates were not read from a primary source; see the label. Blank cells mean the figure was not sourced, not that it is zero.
Trials
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Landmark trials in OnCo
Latest papers
topQuery for this drug: (TITLE:"Nirogacestat" OR ABSTRACT:"Nirogacestat" OR TITLE:"Ogsiveo" OR ABSTRACT:"Ogsiveo") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Nirogacestat, not a curated reading list.