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FDA Grants Approval of TIBSOVO®, the First Oral, Targeted Therapy for Adult Patients with Relapsed/Refractory Acute Myeloid Leukemia and an IH1 Mutation


CAMBRIDGE, Mass., July 20, 2018 (GLOBE NEWSWIRE) -- - Approval of TIBSOVO® was Based on Phase 1 Study Results, Including Rate and Duration of Complete Remission (CR) and CR with Partial Hematologic Recovery (CRh) and Rate of Conversion to Transfusion Independence1 -

- With Second IDHm Inhibitor Approved in Less Than A Year, Treatments Discovered and Developed by Agios Now Available for Relapsed/Refractory AML with an IDH1 or IDH2 Mutation -

- AML Patients with IDH1 and IDH2 Mutations Represent ~20% of All Patients with AML2 -

- Company to Host Investor Conference Call Today at 1p.m. ET -

In July 20, 2018, AGIOS Pharmaceuticals and Inc. (NASDAQ:AGIO) announced that TIBSOVO (ivosidenib) was approved by FDA in the United States to become the first oral drug for targeted treatment for Adult Patients with Relapsed/Refractory Acute Myeloid Leukemia and an IH1 Mutation (R/R AML). 

PharmaBlock Sciences (Nanjing), Inc. has witnessed a series of major milestones of Tibosovo, from research to development and to commercial, as a starting materials supplier of this new drug. PharmaBlock is proud to contribute to the new drug development with expertise in medicinal chemistry and process chemistry. Sincere congratulations to Agios on receiving the second FDA approval within one year.
Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that TIBSOVO® (ivosidenib) was granted approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA approved test. TIBSOVO®, an oral, targeted inhibitor of the IDH1 enzyme, is the first and only FDA-approved therapy for patients with R/R AML and an IDH1 mutation1.

“The FDA approval of TIBSOVO® – our first wholly owned drug and the second approved medicine from our research platform in less than a year – is an incredibly exciting milestone for our company and, importantly, for the approximately 6-10% of AML patients with an IDH1 mutation who have been waiting for new treatment options that work radically different than conventional chemotherapy,” said David Schenkein, M.D., chief executive officer at Agios. “I want to thank the patients and their caregivers, nurses and physicians who participated in our clinical trials. With their support and the dedication of Agios’ employees, we are well on our way to becoming a sustainable multi-product biopharmaceutical company delivering medicines that have the potential to change how serious diseases are treated.”

AML is a cancer of the blood and bone marrow marked by rapid disease progression and is the most common acute leukemia affecting adults with approximately 20,000 new cases estimated in the U.S. each year.3,4 The majority of patients with AML eventually relapse. Relapsed or refractory AML has a poor prognosis.5 The five-year survival rate is approximately 27%.3 For 6 to 10 percent of AML patients, the mutated IDH1 enzyme blocks normal blood stem cell differentiation, contributing to the genesis of acute leukemia.2

“AML patients who relapse or are refractory to available therapies have few, if any, treatment options,” said Hagop M. Kantarjian, M.D., professor and chair of the Department of Leukemia at The University of Texas MD Anderson Cancer Center. “The clinical study demonstrated that TIBSOVO® has the potential to deliver strong, durable responses as a single agent and can help patients achieve and maintain transfusion independence. IDH inhibitors represent a new class of noncytotoxic, targeted therapies for AML patients with IDH mutations.”

TIBSOVO® Safety and Efficacy Data1

The FDA approval was based on the clinical data from an open-label, single-arm, multicenter dose-escalation and expansion trial of adult patients with R/R AML and an IDH1 mutation (Study AG120-C-001, NCT02074839). TIBSOVO® was approved concurrently with the Abbott RealTime? IDH1 companion diagnostic test for selection of patients with R/R AML for treatment with TIBSOVO®. 

The efficacy of TIBSOVO® was evaluated in 174 adult patients with R/R AML with an IDH1 mutation identified or confirmed by the Abbott RealTime? IDH1 assay. TIBSOVO® was given orally at a starting dose of 500 mg daily until disease progression, development of unacceptable toxicity, or undergoing hematopoietic stem cell transplantation. Patients had a median age of 67 years (range of 18 to 87) and 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. The primary endpoint is the combined complete remission (CR) and complete remission with partial hematologic improvement (CRh) rate. CRh is defined as <5% of blasts in the bone marrow, no evidence of disease and partial recovery of peripheral blood counts (platelets >50,000/microliter and ANC >500/microliter).

In this trial, TIBSOVO® demonstrated:

●   CR+CRh rate of 32.8% (57 of 174 patients) (95% CI: 25.8, 40.3).
●   The CR rate was 24.7% (43 of 174 patients) (95% CI 18.5, 31.8) and the CRh rate was 8% (14 of 174 patients) (95% CI 4.5, 13.1).
●   Median duration of CR+CRh was 8.2 months (95% CI: range 5.6, 12 months).
●   For patients who achieved a CR or CRh, the median time to best response of CR or CRh was 2.0 months (range, 0.9 to 5.6 months).
●   Among the 110 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 41 (37.3%) became independent of RBC and platelet transfusions during any 56-day post-baseline period.
●   Of the 64 patients who were independent of both RBC and platelet transfusions at baseline, 38 (59.4%) remained transfusion independent during any 56-day post-baseline period.

●   Twenty-one of the 174 patients (12%) went on to stem cell transplant following TIBSOVO® treatment.

The safety profile of single-agent TIBSOVO® was evaluated in 179 patients with R/R AML with an IDH1 mutation treated with a dose of 500 mg daily. The median duration of exposure to TIBSOVO® was 3.9 months (range 0.1 to 39.5 months). In the clinical trial, 19% (34/179) of patients treated with TIBSOVO® experienced differentiation syndrome, which can be fatal if not treated. QTc interval prolongation and Guillain-Barré Syndrome occurred in patients treated with TIBSOVO®. 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%).

About TIBSOVO® (ivosidenib)

TIBSOVO® (ivosidenib) is an isocitrate dehydrogenase-1 (IDH1) inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. For more information, visit TIBSOVO.com.


1. TIBSOVO® Full Prescribing Information (U.S.). Agios Pharmaceuticals, Inc. Cambridge, MA.
2. DiNardo C. Durable Remissions from Ivosidenib in IDH1-Mutated Relapsed or Refractory AML. New England Journal of Medicine. June 2, 2018
3. National Cancer Institute Surveillance, Epidemiology, and End Results Program. Cancer Stat Facts: Acute Myeloid Leukemia (AML). https://seer.cancer.gov/statfacts/html/amyl.html. Accessed July 2018.
4. American Cancer Society. Acute Myeloid Leukemia (AML). https://www.cancer.org/content/dam/CRC/PDF/Public/8674.00.pdf. Accessed July 2018.
5. Kumar C. Genetic Abnormalities and Challenges in the Treatment of Acute Myeloid Leukemia. Genes Cancer. 2011; 2:95-107.

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