施维雅宣布FDA批准拓舒沃（艾伏尼布片）用于治疗IDH1突变的复发或难治性（R/R）骨髓增生异常综合征（MDS）

On October 24, 2023, Servier announced that the U.S. Food and Drug Administration (FDA) has approved Tosovo® (ivosidenib tablets) for the treatment of patients with relapsed or refractory (R/R) myelodysplastic syndrome (MDS) with isocitrate dehydrogenase 1 (IDH1) mutations. This is Tosuvo’s fifth indication in the field of IDH1-mutated cancers and the first and only targeted therapy approved for this molecularly defined subgroup of patients with relapsed or refractory MDS.

Tosovo is the first and only targeted therapy approved for the treatment of R/RMDS patients with susceptible IDH1 mutations.

Tosovo’s fifth approved indication consolidates Servier’s leadership in the field of mutant IDH inhibition.

“New applications of targeted therapies across IDH-mutated cancers have emerged as a powerful treatment option for patients in this molecularly defined subgroup,” said Amir Fathi, MD, a hematologist, medical oncologist, and expert in myeloid malignancies. "This new indication for myelodysplastic syndromes with relapsed or refractory IDH1 mutations reinforces the importance of mutation testing to guide treatment decisions and potentially improve patient outcomes."

"Tosovo's approval is good news for the MDS community," said Tracey Iraq, executive director of the MDS Foundation. "Previously, there were no approved targeted therapies available for patients with relapsed or refractory MDS harboring IDH1 mutations. We would like to thank the study participants, their families and caregivers, as well as the researchers at Servier and the clinical investigators who participated in this study, for "We help bring new treatment options to patients with significant unmet needs."

Tosuvo was granted Breakthrough Therapy Designation for the treatment of adult patients with relapsed or refractory (R/R) MDS harboring IDH1 mutations and received Priority Review status. Priority review expedites review timelines and is granted to drug applications that, if approved, would provide a significant improvement in effectiveness or safety in treating, diagnosing, or preventing serious conditions.

The FDA also approved the Abbott RealTime IDH1 Assay as a companion diagnostic device for selecting patients suitable for Tosovo® treatment.

About Tosovo (ivonib tablets)

Tosovo is a precision medicine drug that targets a specific type of mutation, namely isocitrate dehydrogenase 1 (IDH1) mutation. Tosovo has been approved for five indications globally, including approvals in the United States, European Union, Australia and China.

In the United States, Tosovo is indicated for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) harboring an IDH1 mutation; as a single agent or in combination with azacitidine for the treatment of newly diagnosed IDH1-mutated AML in adult patients aged ≥75 years or with comorbidities that preclude intensive induction chemotherapy; as a monotherapy for the treatment of adults with IDH1-mutated relapsed or refractory MDS; and for the treatment of patients with previously treated IDH1-mutated cholangiocarcinoma.

Servier has granted CStone Pharmaceuticals the co-exclusive development rights and exclusive commercialization license for Tosovo® in Greater China (including Mainland China, Taiwan, Hong Kong and Macau) and Singapore.

Important safety information and indications for Tosovo for US patients

Indications

Tosovo is suitable for use in the detection of patients with susceptible isocitrate dehydrogenase-1 (IDH1) mutations by FDA-approved detection methods. Patients:

Newly diagnosed acute myeloid leukemia (AML)

In combination with azacitidine or as monotherapy, it is indicated for adult patients with newly diagnosed AML who are 75 years of age and older or who have comorbidities that preclude the use of intensive induction chemotherapy.

Relapsed or refractory AML

For the treatment of adult patients with relapsed or refractory AML.

Relapsed or refractory myelodysplastic syndrome (MDS)

For the treatment of adult patients with relapsed or refractory myelodysplastic syndrome.

Local advanced or metastatic cholangiocarcinoma

For the treatment of adult patients with previously treated locally advanced or metastatic cholangiocarcinoma.