DeFi
The first randomised trial to show a drug controls desmoid tumours, with better pain and function.
PFS HR 0.29; ORR 41% vs 8% (CR 7%); patient-reported pain and physical function improved. NEJM 2023; approval November 2023.
- The treated group had about 71 percent lower chance of the event at any given time (hazard ratio 0.29).
- The absolute difference, how many more people out of 100 were helped, is not reported here.
- This is a surrogate endpoint: it measures the cancer being controlled or absent on scans and tests, which often, but not always, translates into living longer.
- 41 vs 8 out of 100 had their tumour shrink with Nirogacestat compared with Placebo; 33 more per 100.
- Roughly one extra person helped for every 3 treated. That is a rough figure taken from the two percentages, not a guarantee for any one person.
- A response rate counts how many people had their tumours shrink by a set amount; shrinking is encouraging but does not by itself show that people live longer or feel better.
- These results apply to the people the trial enrolled: Progressing desmoid tumours: nirogacestat vs placebo. People in a different situation may not see the same effect.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
142 participants enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Progression-free survivalprimary | Nirogacestat | 70 | — | 0.29 (0.15–0.55) | <0.001 | link |
| Placebo | 72 | — | ||||
| Objective response rate | Nirogacestat | — | 41% | — | — | — |
| Placebo | — | 8% |
Pages like this
not linked directly; found by shared links- CompanySpringWorks Therapeutics (Merck KGaA)
Shares Nirogacestat, Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
- IdeaMandatory national virtual tumour boards for rare and complex cancers
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
- IdeaOne legal framework for pooling rare cancer data across borders
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- IdeaA funded expert second opinion for every new high-stakes or rare cancer diagnosis
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
- IdeaLet adolescents from age 12 into adult trials when the cancer biology is the same
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
- TermFNCLCC grade (soft-tissue sarcoma)
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
- PersonTerry Fox
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
- IdeaPay a prize for rare cancer drugs instead of hoping for a market
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).