Evidence map›Paper›PMID 40590836›Full record

ArticleClinical cancer research : an official journal of the American Association for Cancer Research2025

FDA Approval Summary: Ivosidenib for Treatment of Adult Patients with Relapsed/Refractory Myelodysplastic Syndrome with an IDH1 Mutation.

Ashley C Woods, E Dianne Pulte, Xin Wang, Jonathon Vallejo, Ritu Chadda, Nan Zheng, Javier G Blanco, Sarah E Dorff, Hongshan Li, Jiang Liu and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Clinical cancer research : an official journal of the American Association for Cancer Research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Review
  3. Therapy of high-risk myelodysplastic syndromes.Bone marrow transplantation · 2026
    Review
  4. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

15 authors.

Ashley C WoodsCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0009-0001-4878-5463
E Dianne PulteCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0003-3233-6206
Xin WangCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0009-0003-8408-0529
Jonathon VallejoCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-9700-4037
Ritu ChaddaCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0009-0006-3613-6841
Nan ZhengCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-4475-3712
Javier G BlancoCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0003-3887-0370
Sarah E DorffCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0009-0004-0762-5844
Hongshan LiCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-8792-2774
Jiang LiuCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-5755-3904
Olanrewaju O OkusanyaCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-4134-7882
Richard PazdurOncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-4771-9923
Marc R TheoretOncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0003-4973-5297
R Angelo de ClaroCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0001-8362-1977
Kelly J NorsworthyCenter for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.ORCID 0000-0002-8462-9994

Funding

Intramural FDA HHS FD999999U.S. Food and Drug Administration (FDA)
6 · The paper itself

Abstract

On October 24, 2023, the FDA approved a supplemental application for ivosidenib (Tibsovo; Servier) for the treatment of adult patients with relapsed/refractory (R/R) isocitrate dehydrogenase 1-mutated myelodysplastic syndrome (MDS). The efficacy of ivosidenib in patients with R/R MDS was established in study AG120-C-001, an open-label, single-arm trial, based on the complete remission (CR) + partial remission (PR) rate, duration of CR + PR, and conversion of transfusion dependence to transfusion independence. With a median follow-up of 27.1 months in 18 patients with isocitrate dehydrogenase 1-mutated R/R MDS treated with ivosidenib 500 mg once daily, the CR + PR rate was 39% (95% confidence interval, 17.3-64.3; all CR responses), with a median duration of response not estimable (range, 1.9-80.8+ months). Of the nine patients with transfusion dependence at baseline, six achieved transfusion independence (67%). Serious adverse reactions in ≥5% included differentiation syndrome, fatigue, and rash. The most common (≥20%) adverse reactions were arthralgia, fatigue, cough, diarrhea, decreased appetite, mucositis, myalgia, and pruritus.

Indexed as

Antineoplastic AgentsGlycineIsocitrate DehydrogenaseMutationMyelodysplastic SyndromesNeoplasm Recurrence, LocalPyridinesAdultAgedAged, 80 and overDrug ApprovalFemaleHumansMaleMiddle AgedTreatment OutcomeAntineoplastic AgentsGlycineIDH1 protein, humanIsocitrate DehydrogenaseivosidenibPyridines

Identifiers

PMID40590836
PMCPMC12233196

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.