Approval Based on Data From the Phase 1/2 ‘205 Study

THOUSAND OAKS, CA, USA I August 29, 2018 I Amgen (NASDAQ:AMGN) today announced that the European Commission (EC) has approved an expanded indication for BLINCYTO® (blinatumomab) as monotherapy for the treatment of pediatric patients aged one year or older with Philadelphia chromosome-negative CD19 positive B-cell precursor acute lymphoblastic leukemia (ALL), which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation. The approval is based on results from the Phase 1/2 ‘205 study, an open-label, multicenter, single-arm trial which evaluated the efficacy and safety of BLINCYTO in pediatric patients with relapsed or refractory B-cell precursor ALL.

“Historically, children with relapsed or refractory ALL have had limited pharmacologic options beyond chemotherapy, resulting in poor outcomes,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “This approval for BLINCYTO provides physicians across Europe with an important new immunotherapy option for these young, heavily pretreated patients, delivering on Amgen’s commitment to making a difference in the lives of cancer patients.”

ALL is a rapidly progressing cancer of the blood and bone marrow that occurs in both adults and children.1,2 In Europe, an estimated 5,000 children are diagnosed with ALL each year.3

BLINCYTO is the first-and-only bispecific T cell engager (BiTE®) immunotherapy construct approved globally. It is also the first immunotherapy from Amgen’s BiTE® platform, an innovative approach that helps the body’s immune system target cancer cells.

Approval via the centralized procedure grants a marketing authorization from the EC, which is valid in all European Union (EU) and European Economic Area (EEA)-European Free Trade Association (EFTA) states (Norway, Iceland and Liechtenstein).

About Study ‘205
Study ‘205 evaluated the safety and efficacy of BLINCYTO in a Phase 1/2 open-label, multicenter, single-arm study in 93 pediatric patients with Ph- relapsed or refractory B-cell precursor ALL (second or later bone marrow relapse, any marrow relapse after allogeneic hematopoietic stem cell transplantation [alloHSCT], or refractory to other treatments and had greater than 25 percent blasts in bone marrow). The results were published in the Journal of Clinical Oncology.

BLINCYTO was administered as a continuous intravenous infusion. The recommended dose for this study was determined to be 5 μg/m2/day on days 1-7 and 15 μg/m2/day on days 8-28 for cycle 1, followed by two weeks off, and 15 μg/m2/day on days 1-28, followed by two weeks off for subsequent cycles. Dose adjustment was possible in case of adverse events. Patients who responded to BLINCYTO, but later relapsed, had the option to be retreated with BLINCYTO. 

Among the 70 patients treated at the recommended dosage, the median age was eight years (range: seven months to 17 years), 40 out of 70 (57.1 percent) had undergone alloHSCT prior to receiving BLINCYTO, and 39 out of 70 (55.7 percent) had refractory disease; the mean number of treatment cycles was 1.5. Twenty out of 70 patients (28.6 percent) achieved a complete response or complete response with partial hematologic recovery within two treatment cycles; with 17 of 20 responses (85 percent) occurring within the first cycle. In general, the adverse reactions in BLINCYTO-treated pediatric patients were similar in type to those seen in adult patients with relapsed or refractory B-cell precursor ALL.

About BLINCYTO® (blinatumomab)
BLINCYTO is a bispecific CD19-directed CD3 T cell engager (BiTE®) immunotherapy that binds to CD19 expressed on the surface of cells of B-lineage origin and CD3 expressed on the surface of effector T cells. BLINCYTO was granted breakthrough therapy and priority review designations by the FDA in 2014, and carries full approval in the U.S. for the treatment of relapsed or refractory B-cell precursor ALL in adults and children. In the U.S., BLINCYTO is also approved under accelerated approval for the treatment of adults and children with B-cell precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1 percent.

In 2015, BLINCYTO was approved in the EU for the treatment of adults with Ph- relapsed or refractory B-cell precursor ALL.

About BiTE® Technology
Bispecific T cell engager (BiTE®) antibody constructs are a novel immune-oncology technology that can be engineered to target any tumor antigen expressed by any type of cancer. The modified antibodies are designed to kill malignant cells using the patient’s own immune system by bridging T cells to tumor cells. BiTE® antibody constructs help connect the T cells to the targeted cell, with the intent of causing T cells to inject toxins which trigger cancer cell death (apoptosis). Amgen is developing BiTE® antibody constructs to uniquely (or specifically) target numerous hematologic malignancies and solid tumors.

Important EU BLINCYTO® (blinatumomab) Safety Information

This product is subject to additional monitoring in the EU. All suspected adverse reactions should be reported in accordance with the national reporting system.

BLINCYTO has been evaluated in paediatric patients with relapsed or refractory B-precursor ALL in a phase I/II dose escalation/evaluation study, in which 70 paediatric patients, aged 7 months to 17 years, were treated with the recommended dosage regimen.

The most frequently reported serious adverse events were pyrexia (11.4%), febrile neutropenia (11.4%), cytokine release syndrome (5.7%), sepsis (4.3%), device-related infection (4.3%), overdose (4.3%), convulsion (2.9%), respiratory failure (2.9%), hypoxia (2.9%), pneumonia (2.9%), and multi-organ failure (2.9%).

The adverse reactions in BLINCYTO-treated paediatric patients were similar in type to those seen in adult patients. Adverse reactions that were observed more frequently (≥ 10% difference) in the paediatric population compared to the adult population were anaemia, thrombocytopenia, leukopenia, pyrexia, infusion-related reactions, weight increase, and hypertension.

The type and frequency of adverse events were similar across different paediatric sub-groups (gender, age, geographic region).

Please refer to the Summary of Product Characteristics for full European prescribing information.

Important Safety Information Regarding BLINCYTO® (blinatumomab) U.S. Indication


  • Cytokine Release Syndrome (CRS), which may be life-threatening or fatal, occurred in patients receiving BLINCYTO®. Interrupt or discontinue BLINCYTO® as recommended.
  • Neurological toxicities, which may be severe, life-threatening or fatal, occurred in patients receiving BLINCYTO®. Interrupt or discontinue BLINCYTO® as recommended.

About Amgen’s Commitment to Oncology

Amgen Oncology is committed to helping patients take on some of the toughest cancers, such as those that have been resistant to drugs, those that progress rapidly through the body and those where limited treatment options exist. Amgen’s supportive care treatments help patients combat certain side effects of strong chemotherapy, and our targeted medicines and immunotherapies focus on more than a dozen different malignancies, ranging from blood cancers to solid tumors. With decades of experience providing therapies for cancer patients, Amgen continues to grow its portfolio of innovative and biosimilar oncology medicines.

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About Amgen

Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people’s lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world’s leading independent biotechnology company, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential

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  1. Cancer Research UK. About acute lymphoblastic leukaemia (ALL). Accessed June 6, 2018. 
  2. Mayo Clinic. Acute lymphocytic leukemia. Accessed June 6, 2018.
  3. Conter V, et al. Acute lymphoblastic leukemia. Orphanet Encyclopedia. Accessed Jan. 30, 2018.