FDA approves Tibsovo for treatment of advanced myelodysplastic syndromes
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The FDA approved ivosidenib for treatment of certain adults with relapsed or refractory myelodysplastic syndromes.
The indication applies to use of the agent in adults who harbor susceptible isocitrate dehydrogenase (IDH1) mutations as detected by an FDA-approved companion diagnostic test.
Ivosidenib (Tibsovo, Servier) — an IDH1 inhibitor — is the first targeted therapy approved for the treatment of relapsed or refractory IDH1-mutant MDS and has been previously approved for the several other IDH1-mutant tumors.
“This approval for Tibsivo is welcome news for the MDS community,” Tracey Iraca, executive director of the MDS Foundation, said in a Servier-issued press release.
“Before [this decision], there were no approved targeted therapies available to [patients with relapsed or refractory MDS] harboring the IDH1 mutation,” she added. “We want to thank the study participants, their families and caregivers, as well as the researchers at Servier and clinical investigators involved in this study for helping to bring a new treatment option to patients where there has been a significant unmet need.”
FDA based approval on results from a single-arm phase 1 trial (AG120-C-001; NCT02074839). The open-label multicenter study enrolled 18 adults with relapsed or refractory MDS harboring an IDH1 mutation as determined by local testing from peripheral blood or bone marrow and retrospectively confirmed using the Abbott RealTime IDH1 Assay.
Study participants received daily oral ivosidenib at a dose of 500 mg for 28-day cycles until they experienced disease progression, unacceptable toxicity or underwent hematopoietic stem cell transplantation.
Results showed a complete remission rate of 38.9% (95% CI, 17.3-64.3), with all observed responses noted as complete responses to therapy.
Median duration of response had not been reached as of the study’s data cutoff date (range, 1.9-80.8 months).
Investigators also reported a median time to complete remission of 1.9 months (range, 1 to 5.6).
Six of 9 patients (67%) dependent on red blood cell or platelet transfusions before therapy became transfusion-independent with 56 days of baseline treatment. Meanwhile, 7 of 9 (78%) transfusion-independent patients prior to therapy remained so up to 56 days after baseline treatment with ivosidenib.
The most common adverse events related to the use of ivosidenib included gastrointestinal toxicities, arthralgia, fatigue, cough, myalgia and rash.
Prescribing information for ivosidenib also includes a boxed warning about the risk for life-threatening or fatal differentiation syndrome among patients who receive the therapy for acute myeloid leukemia or MDS.
The FDA also approved the Abbott RealTime IDH1 Assay as a companion diagnostic to determine individuals who are eligible for treatment with ivosidenib.
References:
- FDA approves ivosidenib for myelodysplastic syndromes (press release). Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ivosidenib-myelodysplastic-syndromes?utm_medium=email&utm_source=govdelivery. Published Oct. 24, 2023. Accessed Oct. 25, 2023.
- Servier announces FDA approval of Tibsovo (ivosidenib tablets) for the treatment of IDH1-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS) (press release). Available at: https://servier.us/blog/servier-announces-fda-approval-of-tibsovo-ivosidenib-tablets-for-the-treatment-of-idh1-mutated-relapsed-or-refractory-r-r-myelodysplastic-syndromes-mds/. Published Oct. 24, 2023. Accessed Oct. 25, 2023.
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