Rytelo imetelstat
Drug Class
Telomerase inhibitor端粒酶抑制剂
Mechanism
Binds directly to the RNA component of telomerase (hTR) to inhibit enzymatic activity, suppressing expansion of malignant hematopoietic clones.直接结合端粒酶的 RNA 组分 (hTR) 以抑制其酶活性，从而抑制恶性造血克隆细胞的增殖。
Approved Indication
Treatment of adult patients with low- to intermediate-1-risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or are ineligible for erythropoiesis-stimulating agents (ESAs).用于治疗中低危 (low- to intermediate-1-risk) 骨髓增生异常综合征 (MDS) 伴输血依赖性贫血（8周内需要输注4个或更多单位红细胞），且对促红细胞生成刺激剂 (ESA) 无反应或不适用的成人患者。
What This Means For Patients
Rytelo (imetelstat) is a first-in-class oligonucleotide telomerase inhibitor indicated for patients with lower-risk transfusion-dependent myelodysplastic syndromes (MDS). It targets malignant progenitor cells to restore healthy blood production. It is available under the special-import policy in the Hainan Boao Lecheng pilot zone.伊美司他（Rytelo）是首个寡核苷酸端粒酶抑制剂类药物，适用于中低危输血依赖性骨髓增生异常综合征（MDS）患者的贫血治疗。它靶向恶性祖细胞以恢复正常的造血功能。该药物已通过先行区特许进口政策引入博鳌乐城。
Regulatory Approvals
- FDA — approved (2024) — label
Pivotal Clinical Evidence
- IMerge — Platzbecker et al., Lancet 2024 (NCT02598661) — DOI
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.