TIBSOVO is the first and only approved targeted therapy for R/R MDS patients with a susceptible IDH1 mutation
Fifth approved indication for TIBSOVO solidifies Servier’s leadership in mutant IDH inhibition
BOSTON, MA, USA I October 24, 2023 I 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 approved TIBSOVO® (ivosidenib tablets) for the treatment of patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS). This is the fifth indication for TIBSOVO across IDH1-mutated cancers, and the first and only approved targeted therapy for people diagnosed with R/R MDS within this molecularly defined subset.
“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, Servier Pharmaceuticals. “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 FDA approval of this indication is supported by a pivotal Phase 1, open-label study in IDH1-mutated R/R MDS patients (n=18) where 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 1.9 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.
“The novel use of targeted therapy across IDH-mutated cancers has become a powerful therapeutic option for patients within this molecularly defined subset,” said Amir Fathi, M.D., 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.”
An estimated 16,000 people in the U.S. are diagnosed with MDS each year.1 Approximately 3.6% of MDS patients have an IDH1 mutation,2 which is considered an early “driver” mutation.3 For MDS patients with an IDH1 mutation, the prognosis has often been associated with worse overall outcomes and an increased risk of transformation to AML.2
“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.”
“An MDS diagnosis is ambiguous. I remember feeling confused trying to make sense of my diagnosis, what having an IDH1-mutation meant, and what options were available for my treatment plan,” said Susan, a patient living with IDH1-mutated MDS.** “The news of an FDA approval for a targeted therapy in IDH-1 mutated MDS has given me a tremendous sense of gratitude and provides hope to patients – like me – who are living with this disease. I want to thank everyone who played a role in this major step forward for the MDS community.”
TIBSOVO was granted Breakthrough Therapy designation for the treatment of adult patients with R/R (MDS) with an IDH1 mutation and received Priority Review, which accelerated the review timeline and 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.4
The FDA also approved the Abbott RealTime IDH1 Assay as a companion diagnostic device to select patients for TIBSOVO.
* Denotes a censored observation.
**Last name withheld to protect privacy.
About the NCT02074839 Clinical Trial5
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+PR, duration of transfusion independence, and time to transfusion independence.
About TIBSOVO® (ivosidenib tablets)
TIBSOVO is a precision medicine that targets a specific type of mutation known as isocitrate dehydrogenase 1 (IDH1). TIBSOVO is approved in five indications globally, including approvals in the U.S., European Union, Australia, and China.
In the U.S., TIBSOVO is approved 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, as monotherapy for the treatment of adult patients with IDH1-mutant relapsed or refractory MDS, and for patients with previously treated IDH1-mutated cholangiocarcinoma.
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.
For more information about TIBSOVO in the U.S., please visit www.tibsovo.com.
About Myelodysplastic Syndromes
Myelodysplastic Syndromes (MDS) are disorders in which progenitor (stem) cells do not mature into healthy blood cells.6 In the U.S., approximately 16,000 new cases of MDS are reported each year.1 Approximately 3.6% of MDS patients have an IDH1 mutation,2 which is considered an early “driver” mutation.3 For MDS patients with an IDH1 mutation, the prognosis has often been associated with worse overall outcomes and in an increased risk of transformation to AML.2 Prior to the approval of TIBSOVO, there were no approved targeted therapies for this molecularly defined subset.
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.
INDICATIONS
TIBSOVO is indicated for patients with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test with:
Newly Diagnosed Acute Myeloid Leukemia (AML)
- In combination with azacitidine or as monotherapy for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy
Relapsed or Refractory AML
- For the treatment of adult patients with relapsed or refractory AML
Relapsed or Refractory Myelodysplastic Syndromes (MDS)
For the treatment of adult patients with relapsed or refractory myelodysplastic syndromes
Locally Advanced or Metastatic Cholangiocarcinoma
For the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated
Please see Full Prescribing Information, including BOXED WARNING for AML and MDS patients.
1 SEER MDS Incidence Rates. https://seer.cancer.gov/statistics-network/explorer/application.html?site=409&data_type=1&graph_type=2&compareBy=age_range&chk_age_range_1=1&chk_age_range_16=16&chk_age_range_62=62&chk_age_range_122=122&chk_age_range_160=160&chk_age_range_166=166&hdn_rate_type=1&sex=1&race=1&hdn_stage=101&advopt_precision=1&advopt_show_ci=on&hdn_view=1&advopt_show_apc=on&advopt_display=1; U.S. Census Bureau Population Estimates 2017 National Population Projections Tables: Main Series (census.gov) https://www.census.gov/data/tables/2017/demo/popproj/2017-summary-tables.html.
2 Thol, F., Weissinger, E. M., Krauter, J., Wagner, K., Damm, F., Wichmann, M., Göhring, G., Schumann, C., Bug, G., Ottmann, O., Hofmann, W. K., Schlegelberger, B., Ganser, A., & Heuser, M. (2010). IDH1 mutations in patients with myelodysplastic syndromes are associated with an unfavorable prognosis. Haematologica, 95(10), 1668–1674. https://doi.org/10.3324/haematol.2010.025494. Accessed May 19, 2023.
3 DiNardo CD, et al. Leukemia. 2016;30(4):980-984. doi:10.1038/leu.2015.211.
4 United States Food and Drug Administration (FDA). Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review. Accessed June 27, 2023.
5 Clinicaltrials.gov website. https://clinicaltrials.gov/ct2/show/NCT02074839. Accessed September 1, 2023.
6 Pagliuca, S., Gurnari, C., & Visconte, V. (2021). Molecular Targeted Therapy in Myelodysplastic Syndromes: New Options for Tailored Treatments. Cancers, 13(4), 784. https://doi.org/10.3390/cancers13040784.
SOURCE: Servier Pharmaceuticals
Post Views: 134
TIBSOVO is the first and only approved targeted therapy for R/R MDS patients with a susceptible IDH1 mutation
Fifth approved indication for TIBSOVO solidifies Servier’s leadership in mutant IDH inhibition
BOSTON, MA, USA I October 24, 2023 I 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 approved TIBSOVO® (ivosidenib tablets) for the treatment of patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS). This is the fifth indication for TIBSOVO across IDH1-mutated cancers, and the first and only approved targeted therapy for people diagnosed with R/R MDS within this molecularly defined subset.
“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, Servier Pharmaceuticals. “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 FDA approval of this indication is supported by a pivotal Phase 1, open-label study in IDH1-mutated R/R MDS patients (n=18) where 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 1.9 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.
“The novel use of targeted therapy across IDH-mutated cancers has become a powerful therapeutic option for patients within this molecularly defined subset,” said Amir Fathi, M.D., 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.”
An estimated 16,000 people in the U.S. are diagnosed with MDS each year.1 Approximately 3.6% of MDS patients have an IDH1 mutation,2 which is considered an early “driver” mutation.3 For MDS patients with an IDH1 mutation, the prognosis has often been associated with worse overall outcomes and an increased risk of transformation to AML.2
“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.”
“An MDS diagnosis is ambiguous. I remember feeling confused trying to make sense of my diagnosis, what having an IDH1-mutation meant, and what options were available for my treatment plan,” said Susan, a patient living with IDH1-mutated MDS.** “The news of an FDA approval for a targeted therapy in IDH-1 mutated MDS has given me a tremendous sense of gratitude and provides hope to patients – like me – who are living with this disease. I want to thank everyone who played a role in this major step forward for the MDS community.”
TIBSOVO was granted Breakthrough Therapy designation for the treatment of adult patients with R/R (MDS) with an IDH1 mutation and received Priority Review, which accelerated the review timeline and 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.4
The FDA also approved the Abbott RealTime IDH1 Assay as a companion diagnostic device to select patients for TIBSOVO.
* Denotes a censored observation.
**Last name withheld to protect privacy.
About the NCT02074839 Clinical Trial5
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+PR, duration of transfusion independence, and time to transfusion independence.
About TIBSOVO® (ivosidenib tablets)
TIBSOVO is a precision medicine that targets a specific type of mutation known as isocitrate dehydrogenase 1 (IDH1). TIBSOVO is approved in five indications globally, including approvals in the U.S., European Union, Australia, and China.
In the U.S., TIBSOVO is approved 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, as monotherapy for the treatment of adult patients with IDH1-mutant relapsed or refractory MDS, and for patients with previously treated IDH1-mutated cholangiocarcinoma.
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.
For more information about TIBSOVO in the U.S., please visit www.tibsovo.com.
About Myelodysplastic Syndromes
Myelodysplastic Syndromes (MDS) are disorders in which progenitor (stem) cells do not mature into healthy blood cells.6 In the U.S., approximately 16,000 new cases of MDS are reported each year.1 Approximately 3.6% of MDS patients have an IDH1 mutation,2 which is considered an early “driver” mutation.3 For MDS patients with an IDH1 mutation, the prognosis has often been associated with worse overall outcomes and in an increased risk of transformation to AML.2 Prior to the approval of TIBSOVO, there were no approved targeted therapies for this molecularly defined subset.
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.
INDICATIONS
TIBSOVO is indicated for patients with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test with:
Newly Diagnosed Acute Myeloid Leukemia (AML)
- In combination with azacitidine or as monotherapy for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy
Relapsed or Refractory AML
- For the treatment of adult patients with relapsed or refractory AML
Relapsed or Refractory Myelodysplastic Syndromes (MDS)
For the treatment of adult patients with relapsed or refractory myelodysplastic syndromes
Locally Advanced or Metastatic Cholangiocarcinoma
For the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma who have been previously treated
Please see Full Prescribing Information, including BOXED WARNING for AML and MDS patients.
1 SEER MDS Incidence Rates. https://seer.cancer.gov/statistics-network/explorer/application.html?site=409&data_type=1&graph_type=2&compareBy=age_range&chk_age_range_1=1&chk_age_range_16=16&chk_age_range_62=62&chk_age_range_122=122&chk_age_range_160=160&chk_age_range_166=166&hdn_rate_type=1&sex=1&race=1&hdn_stage=101&advopt_precision=1&advopt_show_ci=on&hdn_view=1&advopt_show_apc=on&advopt_display=1; U.S. Census Bureau Population Estimates 2017 National Population Projections Tables: Main Series (census.gov) https://www.census.gov/data/tables/2017/demo/popproj/2017-summary-tables.html.
2 Thol, F., Weissinger, E. M., Krauter, J., Wagner, K., Damm, F., Wichmann, M., Göhring, G., Schumann, C., Bug, G., Ottmann, O., Hofmann, W. K., Schlegelberger, B., Ganser, A., & Heuser, M. (2010). IDH1 mutations in patients with myelodysplastic syndromes are associated with an unfavorable prognosis. Haematologica, 95(10), 1668–1674. https://doi.org/10.3324/haematol.2010.025494. Accessed May 19, 2023.
3 DiNardo CD, et al. Leukemia. 2016;30(4):980-984. doi:10.1038/leu.2015.211.
4 United States Food and Drug Administration (FDA). Fast Track, Breakthrough Therapy, Accelerated Approval, Priority Review. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/priority-review. Accessed June 27, 2023.
5 Clinicaltrials.gov website. https://clinicaltrials.gov/ct2/show/NCT02074839. Accessed September 1, 2023.
6 Pagliuca, S., Gurnari, C., & Visconte, V. (2021). Molecular Targeted Therapy in Myelodysplastic Syndromes: New Options for Tailored Treatments. Cancers, 13(4), 784. https://doi.org/10.3390/cancers13040784.
SOURCE: Servier Pharmaceuticals
Post Views: 134
