Posted on

FDA OKs Targeted Agent Ivosidenib for Relapsed/Refractory AML

Posted on

The US Food and Drug Administration (FDA) has approved the targeted agent ivosidenib (Tibsovo, Agios Pharmaceuticals) for adults with relapsed or refractory acute myeloid leukemia (AML) who harbor isocitrate dehydrogenase-1 (IDH1) mutations.

At the same time, the FDA has also approved the RealTime IDH1 Assay (Abbott Laboratories), a companion diagnostic used to detect specific mutations in the IDH1 gene in blood or bone marrow samples from patients with AML.

Tibsovo is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH1 mutation,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

Padzur added in a statement from the agency that the use of Tibsovo is “associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions.”

Mutations in the gene encoding IDH1 occur in approximately 6% to 10% of patients with AML. Ivosidenib, a first-in-class agent, is an oral, targeted, small-molecule inhibitor of mutant IDH1.

Last year the FDA approved enasidenib (Idhifa, Celgene),  an oral targeted inhibitor of the IDH2 enzyme for treatment of relapsed or refractory AML with an IDH2 mutation. Similarly, it was also approved for use with a companion diagnostic, the RealTime IDH2 Assay (Abbott Laboratories).

The new approval was based on results of a single-arm trial that included 174 adult patients with relapsed or refractory AML and an IDH1 mutation. Patients had a median age of 67 years (range, 18 to 87 years) and had received a median of two prior anticancer therapies (ranging from one to six). More than half (63%) were refractory to previous therapy and 33% had secondary AML.

Among 110 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 41 (37.3%) achieved transfusion independence during any 56-day postbaseline period.

Of the 64 patients who were independent of both RBC and platelet transfusions at baseline, 38 (59.4%) remained transfusion independent during the same period.

In addition, 21 patients were able to become eligible for stem cell transplant following treatment.

The safety profile was evaluated in 179 patients who were treated with a dose of 500 mg daily, and the median duration of exposure was 3.9 months (range, 0.1 to 39.5 months). Of this group, 19% (34/179)  experienced potentially fatal differentiation syndrome; prolonged corrected QT interval and Guillain-Barré syndrome also occurred.

The most common adverse reactions (≥20%) of any grade were fatigue, leukocytosis, arthralgia, diarrhea, dyspnea, edema, nausea, mucositis, electrocardiogram QT prolonged, rash, pyrexia, cough, and constipation. The most frequent serious adverse reactions (≥5%) were differentiation syndrome (10%), leukocytosis (10%) and electrocardiogram QT prolonged (7%).

Ivosidenib was granted Fast Track and Priority Review designations and also received Orphan Drug designation.

GDMeds, an India Pharmacy Service company

[Disclaimer]The Site is operated by GDMeds and all rights thereto are owned and reserved by GDMeds. The contents and works on these pages compiled by GDMeds are subject to copyright law. Copying, processing, distribution and any kind of use outside the limits of copyright law require the written consent of GDMeds In case the content is not created by GDMeds the copyrights of third parties are being observed. However, if a user becomes aware of a copyright infringement, GDMeds asks the user for notification. Upon notification of such violations, GDMeds will remove the content immediately.

[免责声明]本网站由印度极得美运营。印度极得美拥有和保留一切权利。印度极得美的网页内容及文档受版权法保护。复制、加工、传播及任何超出版权法限制的任何使用行为均必须得到印度极得美书面许可。如相关内容如非由印度极得美创作,需遵守第三方的版权,特别是标示为第三方内容的。如用户发现侵犯版权的行为,请通知极得美。一旦收到违反通知,极得美将立刻移除相关内容。

[отказ]Сайт управляется GDMeds, и все права на него принадлежат и зарезервированы GDMeds. Содержание и работы на этих страницах, составленные GDMeds, защищены законом об авторском праве. Копирование, обработка, распространение и любое использование вне пределов авторского права требуют письменного согласия GDMeds. Если контент не создан GDMeds, то соблюдаются авторские права третьих лиц. Однако, если пользователь узнает о нарушении авторских прав, GDMeds запрашивает у пользователя уведомление. После уведомления о таких нарушениях GDMeds немедленно удалит контент.