Bristol Myers Squibb Receives Positive CHMP Opinion for Onureg® (azacitidine tablets; CC-486) as Frontline Oral Maintenance Therapy for Adults with Acute Myeloid Leukemia in First Remission

If approved, Onureg will become the first and only once daily oral frontline maintenance therapy in Europe for patients with a broad range of acute myeloid leukemia (AML) subtypes

In the pivotal QUAZAR® AML-001 study, Onureg demonstrated significant overall survival and showed a relapse-free survival benefit in patients with AML in first remission

PRINCETON, NJ, USA I April 23, 2021 IBristol Myers Squibb (NYSE: BMY) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Onureg® (azacitidine tablets; CC-486) as a maintenance therapy in adult patients with acute myeloid leukemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT).

The CHMP recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, Onureg will be the first and only once daily frontline oral maintenance therapy to demonstrate significant overall survival in patients with a broad range of AML subtypes in first remission.

The CHMP adopted a positive opinion based on results from the QUAZAR® AML-001 study, a Phase 3, international, randomized, double-blind trial. Eligible patients were ages 55 years or older, had newly diagnosed AML, intermediate or poor cytogenetics, had achieved first CR or CRi following intensive induction chemotherapy with or without consolidation treatment (per investigator preference prior to study entry), and were not candidates for HSCT at the time of screening.1

“Maintenance therapy options for acute myeloid leukemia that prolong overall survival have been urgently needed in Europe, especially oral options that can be taken at home. While many patients with acute myeloid leukemia achieve remission with induction therapy, responses to treatment may be of short duration and the risk of relapse remains high, especially for patients who are not eligible for stem cell transplant,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb. “We look forward to the European Commission’s decision and to making Onureg available to appropriate patients, building on our commitment to deliver innovative therapies that improve long-term outcomes for patients.”

The EC is expected to deliver its final decision within 67 days of receipt of the CHMP opinion. The decision will be applicable to all EU member states and Iceland, Norway and Liechtenstein.*

Onureg is approved in the United States for the continued treatment of adult patients with AML who achieved first CR or CRi following intensive induction chemotherapy and who are not able to complete intensive curative therapy.2 In Canada, Onureg is approved as a maintenance therapy for adult patients with AML who achieved CR or CRi following induction therapy with or without consolidation treatment, and who are not eligible for HSCT.3

*Centralized Marketing Authorization does not include approval in Great Britain (England, Scotland and Wales).

About QUAZAR® AML-001

QUAZAR® AML-001, is a Phase 3, international, randomized, double-blind study. Eligible patients were ages 55 years or older, had newly diagnosed AML, intermediate or poor cytogenetics, had achieved first CR or CRi following intensive induction chemotherapy with or without consolidation treatment (per investigator preference prior to study entry) within four months before randomization, and were not candidates for HSCT at the time of screening. The study enrolled 472 patients, randomized 1:1 to receive either Onureg 300 mg (N=238) or placebo (N=234) orally, once daily, for 14 days of a 28-day cycle, plus best supportive care.1

Median OS, the primary endpoint, from time of randomization was greater than two years (24.7 months; 95% CI: 18.7 to 30.5) in the Onureg arm compared to 14.8 months for placebo (HR: 0.69, 95% CI: 0.55 to 0.86; p=0.0009). A subgroup analysis showed consistency in the OS benefit for patients in either CR or CRi. The median duration of treatment was 12 cycles (1 to 82) for Onureg1 and 6 cycles with placebo (1 to 76).4

Serious adverse reactions occurred in 15% of patients who received Onureg. Serious adverse reactions in ≥2% of patients who received Onureg included pneumonia (8%) and febrile neutropenia (7%). One fatal adverse reaction (sepsis) occurred in a patient who received Onureg. The most common adverse reactions with Onureg versus placebo were nausea (65%, 24%), vomiting (60%, 10%), diarrhea (50%, 21%), fatigue/asthenia (44%, 25%), constipation (39%, 24%), pneumonia (27%, 17%), abdominal pain (22%, 13%) arthralgia (14%, 10%), decreased appetite (13%, 6%), febrile neutropenia (12%, 8%), dizziness (11%, 9%) and pain in extremity (11%, 5%). Of patients who received Onureg, permanent discontinuation due to an adverse reaction occurred in 8% of patients.

About AML

AML is one of the most common acute leukemias in adults.5 The worldwide incidence of AML has been estimated at more than 350,000 cases, and the estimated 5-year survival rate for AML in Europe is 17%.6 AML is characterized by the rapid growth of abnormal cells in the bone marrow and as such interferes with normal blood cell production and function. Because of the impaired production of red blood cells, platelets and white blood cells, it can present with signs of anemia, bleeding and infections.5 AML is a heterogeneous disease associated with diverse genetic mutations, and can rapidly progress and lead to death if not promptly treated. 7

AML response to treatment may be of short duration, meaning following patients' initial response to chemotherapy, there is still a very high risk of relapse, thus representing a significant unmet need for maintenance treatment options that prolong overall survival. 8

About Onureg ®

Onureg, the first and only FDA-approved continued AML treatment for patients in first remission, is a once daily oral hypomethylating agent that incorporates into DNA and RNA. The main mechanism of action is thought to be hypomethylation of DNA, as well as direct cytotoxicity to abnormal hematopoietic cells in the bone marrow. Hypomethylation may restore normal function to genes that are critical for cell differentiation and proliferation.9,10 Onureg is approved in the U.S. for continued treatment of adult patients with AML who achieved first CR or CRi following intensive induction chemotherapy and are not able to complete intensive curative therapy. Onureg is also approved in Canada as maintenance therapy for adult patients with AML who achieved CR or CRi following induction therapy with or without consolidation treatment, and who are not eligible for HSCT.

Please see full Prescribing Information for ONUREG.

Bristol Myers Squibb: Creating a Better Future for People with Cancer

Bristol Myers Squibb is inspired by a single vision—transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.

Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.

References:

  1. Clinical Trials.gov. Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia in Complete Remission (QUAZAR AML-001). Available at https://clinicaltrials.gov/ct2/show/NCT01757535. Accessed February 2021.
  2. ONUREG U.S. Prescribing Information. Accessed February 2021.
  3. ONUREG Canada Product Monograph. Accessed January 2021.
  4. Data on File. Princeton, NJ: Bristol-Myers Squibb Company. 2020.
  5. American Cancer Society. What is AML?. https://www.cancer.org/cancer/acute-myeloid-leukemia/about/what-is-aml.html. Accessed on: July 23, 2020.
  6. Maynadie et al. Haematologica. 2013 Feb; 98(2): 230–238.
  7. Int J Hematol Oncol Stem Cell Res. Acute Myeloid Leukemia—Genetic Alterations and Their Clinical Prognosis. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5767295/.
  8. Leukaemia Care. Relapse in Acute Myeloid Leukaemia (AML). https://media.leukaemiacare.org.uk/wp-content/uploads/Relapse-in-Acute-Myeloid-Leukaemia-AML-Web-Version.pdf. Accessed on July 23, 2020.
  9. Laille et al. PLoS One. 2015;10(8):e0135520
  10. Garcia-Manero et al. J Clin Oncol. 2011;29(18):2521–7

SOURCE: Bristol Myers Squibb

La Merie Biologics

FREE Weekly News Bulletin

Sign Up

2020 Sales ofAntibodies & Proteins

New Product Alert

For La Merie Publishing

Sign Up

Top