Servier Announces FDA Filing Acceptance and Priority Review for TIBSOVO® (ivosidenib tablets) in the Treatment of IDH1-mutated Relapsed or Refractory (R/R) Myelodysplastic Syndromes (MDS)
Servier Announces FDA Filing Acceptance and Priority Review for TIBSOVO® (ivosidenib tablets) in the Treatment of IDH1-mutated Relapsed or Refractory (R/R) Myelodysplastic Syndromes (MDS)
If approved, TIBSOVO will be the first and only approved targeted therapy for MDS patients with a susceptible IDH1 mutation
Submission supported by comprehensive clinical data package, including updated results that demonstrate durable remissions and an acceptable safety profile
BOSTON, August 15, 2023 – Servier, a leader in oncology committed to bringing the promise of tomorrow to the patients we serve, today announced the U.S. Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) and granted Priority Review for TIBSOVO® (ivosidenib tablets) in the treatment of patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS). If approved, TIBSOVO would be a first-in-class targeted therapy option for MDS patients within this molecularly defined subset.
The sNDA is based on key results from a pivotal Phase 1, open-label study in IDH1-mutated R/R MDS patients, in which TIBSOVO demonstrated durable remissions, including complete response in nearly 40% of patients, and an acceptable safety profile. The updated efficacy results were recently presented at the 2023 European Hematology Association (EHA) Congress.
These findings showed that in the efficacy analysis set (n=18), a complete remission (CR) rate of 38.9% and objective response rate (ORR) of 83.3% were documented in patients treated with TIBSOVO. In addition, the median time to CR was 1.87 months (range: 1.0, 5.6). At the time of data cutoff, the median duration of CR had not been reached (range: 1.9, 80.8*) and the median overall survival was 35.7 months (range: 3.7*, 88.7*). Additionally, of the nine patients who were transfusion dependent with red blood cells or platelets at baseline, 66.7% (n=6) became independent of transfusions during any ≥56-day post-baseline period. Overall, treatment-related adverse events were consistent with the known safety profile of TIBSOVO. There were no new safety signals identified in this study.
“While the novel use of targeted IDH inhibition has been proven across a number of difficult-to-treat cancers, there continues to be a significant unmet need for MDS patients within this molecularly defined subset, especially for those who experience disease progression,” said Amir Fathi, M.D., Program Director, Center for Leukemia at Massachusetts General Hospital and Associate Professor of Medicine at Harvard Medical School. “Today’s filing acceptance provides further support for the potential efficacy and acceptable safety profile of TIBSOVO in relapsed or refractory MDS and reinforces the importance of mutational testing in this patient population.”
Priority Review is granted to applications for medicines that, if approved, would provide significant improvements in the effectiveness or safety of the treatment, diagnosis, or prevention of serious conditions.[i]
FDA Breakthrough Therapy designation was previously granted for TIBSOVO for the treatment of adult patients with R/R MDS with a susceptible IDH1 mutation as detected by an FDA-approved test.
* Denotes a censored observation.
About the NCT02074839 Clinical Trial[ii]
This Phase I, open-label multinational study evaluated the safety, tolerability, and clinical activity of ivosidenib in patients with relapsed or refractory myelodysplastic syndromes with an IDH1 mutation. The primary endpoint was complete remission (CR) plus partial remission (PR) rate and key secondary endpoints included duration of CR plus PR, duration of transfusion independence, and time to transfusion independence.
About TIBSOVO® (ivosidenib tablets)
TIBSOVO is currently approved in the U.S. as monotherapy for the treatment of adults with IDH1-mutant relapsed or refractory AML and in monotherapy or in combination with azacitidine for adults with newly diagnosed IDH1-mutant AML who are ≥75 years old or who have comorbidities that preclude the use of intensive induction chemotherapy. TIBSOVO was recently approved by the European Commission as a targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy; as well as in monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. TIBSOVO has also been approved in the U.S. and Australia for patients with previously treated IDH1-mutated cholangiocarcinoma. TIBSOVO is also approved in China for the treatment of adult patients with relapsed or refractory AML who have a susceptible IDH1 mutation. Servier has granted CStone a co-exclusive license for the development and an exclusive license agreement for the commercialization of TIBSOVO in Mainland China, Taiwan, Hong Kong, Macao and Singapore.
About Myelodysplastic Syndromes
Myelodysplastic Syndromes (MDS) are disorders in which progenitor (stem) cells do not mature into healthy blood cells.[iii] In the U.S., approximately 16,000 new cases of MDS are reported each year,[iv] and one-third of those MDS patients will eventually progress to acute myeloid leukemia (AML).3 Approximately 3.6% of MDS patients have an IDH1 mutation, which have often been associated with worse overall outcomes and are considered early “driver” mutations in the progression of MDS to AML.[v],[vi] Chemotherapy, supportive therapy, stem cell transplant, growth factors and similar medicines are used to treat MDS.[vii]
About Servier in Oncology
Servier is a global leader in oncology focused on delivering meaningful therapeutic progress for the patients it serves. Governed by a non-profit foundation, Servier approaches innovation with a long-term vision, free of influence from investors and outside pressure to chase short-term monetary targets.
As a leader in oncology, Servier has significantly accelerated its investment in difficult and hard-to-treat cancers, with more than 50% of its research and development dedicated to delivering significant advances in areas of high unmet need throughout oncology with the potential to change the lives of the patients it serves. Within these areas, Servier is the leader in mutant IDH inhibition, with the first ever mutant IDH1 inhibitor approved in the U.S. and the European Union, and the company continues to drive the science behind targeted mutant IDH inhibition.
Servier’s commitment to therapeutic progress guides its collaboration strategy. While many companies across the industry are scaling back investments, Servier is actively building alliances, completing acquisitions, conducting licensing deals and entering new partnerships that can help to accelerate access to therapies for patients in need. With the company’s commercial expertise, global reach, scientific expertise and commitment to clinical excellence, Servier is dedicated to bringing the promise of tomorrow to the patients it serves.
Press Contact
Servier Pharmaceuticals
Julia Ferreira
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