Ogsiveo(nirogacestat)
Oncology· 292 cited findings· 1 source document
Company: Merck B V
What the regulatory reviews say
Ogsiveo (nirogacestat) is a gamma-secretase inhibitor. The pivotal study, NIR-DT-301 (DeFi), evaluated its efficacy with a primary endpoint of progression-free survival (PFS). In the overall population, Ogsiveo demonstrated a PFS Hazard Ratio of 0.29 (95% CI: 0.15, 0.55; p-value < 0.001) compared to placebo. For treatment-naive patients, the objective response rate (ORR) was 50% (95% CI: 26.0, 74.0) for nirogacestat versus 7% (95% CI: 0.2, 33.9) for placebo. Radiographic progression was observed in 11 patients receiving nirogacestat compared to 29 in the placebo group.
Safety data from the NIR-DT-301 study indicated a discontinuation rate due to adverse events of 20% in the nirogacestat arm, compared to 1% in the placebo arm. The drug received an orphan medicinal product designation on 17 October 2019, and a CHMP positive opinion for marketing authorization was issued on 19 June 2025. Concomitant use with moderate or strong CYP3A4 inducers and moderate CYP3A4 inhibitors should be avoided.
Key figures — cited to the source page
| PFS HR (overall population) | 0.29 | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.94 |
| PFS HR 95% CI (overall population) | 0.15, 0.55 | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.94 |
| PFS p-value (overall population) | < 0.001 | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.94 |
| ORR (nirogacestat, treatment naive) | 50 % | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.94 |
| ORR (placebo, treatment naive) | 7 % | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.94 |
| Discontinuation rate due to adverse event (nirogacestat arm) | 20 % | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.106 |
| Discontinuation rate due to adverse event (placebo arm) | 1 % | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.106 |
| CHMP positive opinion date for marketing authorization | 19 June 2025 | Merck B V — Ogsiveo EMA EPAR public assessment report (EMEA/, p.8 |
Source documents
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Figures are as stated in the cited regulatory review documents (different drugs are studied in different trials — values are not head-to-head comparisons). Educational reference only — not medical or investment advice.