FDA Approves Tibsovo for Patients with R/R Myelodysplastic Syndromes

The Food and Drug Administration (FDA) has approved Tibsovo (ivosidenib) for adult patients with relapsed or refractory myelodysplastic syndromes (MDS) who have a susceptible isocitrate dehydrogenase-1 (IDH1) mutation.

Along with Tibsovo, the Abbott RealTime IDH1 Assay diagnostic device was also approved by the FDA to help select patients for treatment with Tibsovo.

The respective approval was based on the phase 1 AG120-C-001 trial, which included 18 adult patients with MDS and an IDH1 mutation. These mutations were detected in peripheral blood or bone marrow via a local or central diagnostic test, which was confirmed by the Abbott RealTime IDH1 Assay.

“Servier is proud to lead the way in mutant IDH inhibition through continued innovations that support patients living with difficult and hard-to-treat cancers,” said Arjun Prasad, head of commercial at Servier Pharmaceuticals, the manufacturer of the drug. “As the first and only targeted therapy available for patients with IDH1-mutated relapsed or refractory myelodysplastic syndromes, today’s FDA approval for Tibsovo reinforces our commitment to deliver significant advances in areas of high unmet need and bring the right treatment, to the right patient, at the right time.”

The median treatment time was 9.3 months in the trial, and one patient underwent a stem cell transplantation after receiving Tibsovo. The median overall survival (time from treatment until death of any cause) was 35.7 months, with an objective response rate (the percentage of patients whose disease shrinks or disappears) of 83.3%.

From the data, the efficacy was determined by the rate of complete remission and partial remission, the duration of both remissions and conversion rate from transfusion dependence to independence. The median time-to-complete remission was 1.9 months.

From the nine patients that were dependent on red blood cells and/or platelet transfusions at baseline, researchers found that six patients became red blood cell and platelet transfusion independent during any 56-day post baseline period.

READ MORE: FDA to Speed Up Review of Tibsovo for IDH-1 Mutant MDA

“The novel use of targeted therapy across IDH-mutated cancers has become a powerful therapeutic option for patients within this molecularly defined subset,” said Dr. Amir Fathi, hematologist, medical oncologist and expert in myeloid malignancies. “This new indication in IDH1-mutated relapsed or refractory myelodysplastic syndromes reinforces the importance of mutational testing to inform treatment decisions and potentially improve patient outcomes.”

The treatment-related side effects were consistent with the already known safety profile of Tibsovo. Common side effects were also similar to the common side effects of TIbsovo monotherapy for acute myeloid leukemia. These side effects included GI toxicities — namely diarrhea, constipation, mucositis and nausea — along with arthralgia, fatigue, cough, myalgia and rash.

“This approval for Tibsovo is welcome news for the MDS community,” said Tracey Iraca, executive director, MDS Foundation. “Before today, there were no approved targeted therapies available to relapsed or refractory MDS patients harboring the IDH1-mutation. 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.”

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