OGSIVEO nirogacestat
Drug Class
Gamma-secretase inhibitor伽马分泌酶抑制剂
Mechanism
Inhibits gamma-secretase activity to block cleavage of Notch receptors, preventing activation of Notch signaling pathways that promote desmoid tumor proliferation.选择性阻断伽马分泌酶的催化活性，从而防止 Notch 受体的裂解与激活，阻断促进硬纤维瘤过度增殖的下游信号传导。
Approved Indication
FDA-approved for adult patients with progressing desmoid tumors who require systemic treatment.FDA批准用于治疗病情进展、且需要系统性治疗的硬纤维瘤成人患者。
What This Means For Patients
OGSIVEO is a novel small-molecule gamma-secretase inhibitor for desmoid tumors. It is approved by the FDA and clinically accessible to patients inside Hainan Boao Lecheng.OGSIVEO 是一种新型口服小分子伽马分泌酶抑制剂，专用于治疗晚期硬纤维瘤。其已获 FDA 批准，可在海南博鳌乐城先行区特许通道申请使用。
Regulatory Approvals
- FDA — approved (2023) — source
Pivotal Clinical Evidence
- DeFi — Gounder et al., N Engl J Med 2023 (NCT03785964) — DOI
Sources
This page provides regulatory and mechanism-of-action information for reference only. It is not medical advice, a treatment recommendation, or a guarantee of outcome. Availability is subject to individual clinical review.