Drug
Drug 6 June 2024
FDA approves imetelstat for transfusion-dependent lower-risk MDS
The FDA approved imetelstat (Rytelo) for adults with low- to intermediate-1 risk MDS and transfusion-dependent anaemia, defined as at least 4 red-cell units over 8 weeks, who have not responded to, have lost response to, or are ineligible for an ESA. In IMerge (NCT02598661), 178 patients were randomised 2:1 to imetelstat or placebo. Red-cell transfusion independence for at least 8 weeks was 39.8% (95% CI 30.9–49.3) versus 15% (7.1–26.6), p<0.001. Independence for at least 24 weeks was 28% versus 3.3%, p<0.001. The labeled dose on this page is 7.1 mg/kg intravenously over 2 hours every 4 weeks.
Source: FDA approval announcement, imetelstat
Drug Authorised 7 March 2025
EMA authorises Rytelo (imetelstat)
The EMA product page lists a marketing authorisation dated 7 March 2025, after a CHMP opinion on 12 December 2024. The indication stated there is transfusion-dependent anaemia in adults with low- to intermediate-1 risk MDS who failed, lost response to, or are ineligible for ESAs. The overview also describes non-isolated del(5q), very low to intermediate risk, regular transfusions, and erythropoietin that is inadequate or unsuitable. The main study figure on that page is 36 of 118 (30.5%) versus 6 of 60 (10%) with no transfusion for at least 8 weeks. That is not the same statistic as the FDA 39.8% versus 15% figure above, and this page does not treat them as the same result.
Source: EMA EPAR, Rytelo
Drug 28 August 2023
Company announcement: FDA expands luspatercept to ESA-naive lower-risk MDS
Bristol Myers Squibb announced that the FDA approved Reblozyl (luspatercept-aamt) for anaemia without previous ESA use in adults with very low- to intermediate-risk MDS who may require regular red-cell transfusions. The company said this was based on interim COMMANDS (NCT03682536) results and expanded the population to ESA-naive patients regardless of ring sideroblast status. At the interim analysis, 58.5% (86 patients) versus 31.2% (48 patients) met red-cell transfusion independence for at least 12 weeks with a mean haemoglobin rise of at least 1.5 g/dL within the first 24 weeks (p<0.0001). This row uses the company announcement. The FDA approval letter was not opened.
Source: Bristol Myers Squibb, 28 August 2023
Drug Decision 27 August 2021 · ARTG 30 August 2021
Australia: luspatercept registered for MDS with ring sideroblasts
The TGA AusPMD page for Reblozyl records a decision on 27 August 2021 and ARTG start on 30 August 2021. The indication on that page is adults with transfusion-dependent anaemia, at least 2 red-cell units over 8 weeks, due to very low-, low-, or intermediate-risk MDS with ring sideroblasts. It is not a substitute for immediate transfusion. This is older than the review window. It is included because the US expansion above is broader than the Australian wording on the page that was opened. A later TGA variation, and a current PBS listing, were not verified.
Source: TGA AusPMD, Reblozyl
Drug 24 October 2023
FDA approves ivosidenib for relapsed or refractory MDS with an IDH1 mutation
The FDA approved ivosidenib (Tibsovo) for relapsed or refractory MDS with a susceptible IDH1 mutation, with the Abbott RealTime IDH1 Assay as companion diagnostic. In AG120-C-001, 18 patients were evaluable. The complete remission rate was 38.9% (95% CI 17.3–64.3). All responses were complete remissions. Median time to complete remission was 1.9 months. The page carries a boxed warning for differentiation syndrome. No later MDS approval for this drug was opened.
Source: FDA approval announcement, ivosidenib
Drug 16 June 2025
VERONA: venetoclax plus azacitidine misses overall survival
AbbVie reported that phase 3 VERONA (NCT04401748), venetoclax plus azacitidine versus placebo plus azacitidine in newly diagnosed higher-risk MDS, did not meet overall survival. The hazard ratio was 0.908 and the stratified log-rank p value was 0.3772. The company said there were no new safety signals and that the result does not change approved venetoclax indications.
Source: AbbVie, 16 June 2025
Drug ASH 2025 · Dana-Farber post 6 December 2025
VERONA at ASH 2025: more responses, no survival gain, subgroups not conclusive
A congress write-up of the ASH 2025 subgroup presentation (Garcia-Manero and colleagues, abstract 235) reports 509 randomised patients, median follow-up 41.2 months, median overall survival 22.2 versus 21.7 months, hazard ratio 0.908 (95% CI 0.733–1.126), p=0.38. Modified overall response was 76.2% versus 57.7% (p<0.0001). More patients given venetoclax proceeded to transplant (16.8% versus 13.0%). Grade greater than 3 treatment-related neutropenia was 77% versus 60%, and thrombocytopenia 66% versus 59%. The write-up describes favourable trends in younger patients, higher blasts, higher IPSS-R, and some mutations.
Dana-Farber’s 6 December 2025 newsroom post, on the same trial of about 500 treatment-naive intermediate, high, or very high risk patients, says there was no overall survival difference across subgroups. It reports higher overall response and red-cell and platelet transfusion independence, febrile neutropenia 23% versus 16%, and post-study transplant in 17% (median 5.6 months) versus 13% (median 6.7 months). It says the subgroup trends were not significant, the subgroups were too small to conclude, and the combination does not blanket-replace azacitidine or decitabine for all intermediate or higher-risk MDS.
Sources: Congress Update in Hematology, ASH 2025 · Dana-Farber, posted 6 December 2025
Drug MD Anderson post 7 December 2025 · ASH 2025 abstract 487
Five-day azacitidine looked better than shorter schedules in lower-risk MDS
MD Anderson reported an ASH 2025 analysis (Ian Bouligny) of 247 people with lower-risk MDS, 151 transfusion-dependent and 96 transfusion-independent, treated with 3-day decitabine, 3-day azacitidine, or 5-day azacitidine. In transfusion-dependent patients, 5-day azacitidine had the best event-free survival versus 3-day azacitidine and better overall survival than both shorter schedules. After baseline adjustment, event-free and overall survival were better. Overall response on the 5-day regimen was 48% if transfusion-dependent and 70% if transfusion-independent. Toxicity was not increased versus the 3-day arms. The post says oral azacitidine-cedazuridine is under investigation. That sentence is not a result. This is an institution news report of a meeting abstract, not a label change.
Source: MD Anderson research news, 7 December 2025
Drug 21 July 2023
ENHANCE: magrolimab plus azacitidine stopped for futility
Gilead stopped phase 3 ENHANCE, magrolimab plus azacitidine versus azacitidine in higher-risk MDS, for futility after a planned analysis. The study had enrolled more than 500 patients. Primary endpoints were complete remission and overall survival. Gilead recommended stopping magrolimab in people with MDS. This is just before the 2024 window. It is included because no later source opened here establishes a CD47 antibody as a higher-risk MDS treatment.
Source: Gilead, 21 July 2023
Drug ANZCTR record last updated 14 July 2026
IMpress: Australian phase 2 of imetelstat after a hypomethylating agent, no efficacy result posted
ACTRN12625000226404 (AMLM27 / IMpress_001) is an open-label phase 2 of imetelstat in higher-risk MDS or AML that has failed hypomethylating-agent therapy, linked by the registry to NCT05583552. The record was registered 28 March 2025 and last updated 14 July 2026. Recruitment status is completed. Last participant enrolment is listed as 11 November 2024. Last data collection is anticipated as 1 June 2026. The page says that after the interim analysis no responder by the primary endpoint definition was identified, and that the study continued in an exploratory way with a change in dose frequency. The sample-size fields show a target of 6 and a final of 7, while the statistics section describes a Simon two-stage plan of up to about 46. This page does not treat either figure as an efficacy result. No response rate is published on the record.
Source: ANZCTR ACTRN12625000226404