NICE Recommends Amgen's BLINCYTO® ▼ (Blinatumomab) for Adults With Relapsed or Refractory Philadelphia Chromosome-Negative B-Precursor Acute Lymphoblastic Leukaemia

CAMBRIDGE, England, April 27, 2017 /PRNewswire/ --

• National Institute for Health and Care Excellence (NICE) Published a Final Appraisal Determination (FAD) in a Single Technology Appraisal Reviewing BLINCYTO (blinatumomab)
• FAD Reinforces Significant Need for Innovative Treatment Options for Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia (ALL)
• Blinatumomab is the First-and-Only Approved Bispecific CD19-Directed CD3 T Cell Engager (BiTE^®) Immunotherapy

Today the National Institute for Health and Care Excellence (NICE) has published its Final Appraisal Determination (FAD) recommending BLINCYTO^® (blinatumomab) as an option for treating adults with Philadelphia-chromosome-negative (Ph-) relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL), on the basis of the discount agreed in the patient access scheme. NICE reviewed the data available on the clinical and cost-effectiveness of blinatumomab. Having considered evidence on the nature of the disease and the value placed on the benefits of blinatumomab by people with the condition, those who represent them, and clinical experts, NICE concluded that it is a cost-effective use of NHS resources under the end-of-life criteria.^[1]

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"Living with ALL, particularly when first line treatment has failed, can have a profound impact on the person's physical and psychological wellbeing. It can also place a big emotional strain on their families and friends," said Zack Pemberton-Whiteley from Leukaemia CARE. "For patients, the knowledge that there are alternative treatments available can provide some relief. We are pleased that NICE has recommended blinatumomab to be made routinely available to patients in England and Wales. This is consistent with a previous decision from the Scottish Medicines Consortium, ending the variations in access between England and Scotland."

Blinatumomab is a bispecific CD19-directed CD3 T cell engager (BiTE^®) antibody construct. It is the first bispecific antibody construct from Amgen's BiTE^® platform, which helps the body's immune system target cancer cells and represents an entirely new area of authorised oncology therapeutics. BiTE^® antibody constructs are currently being investigated for their potential to treat a wide variety of cancers.

"I am absolutely delighted that blinatumomab will be available for this population of patients with ALL. It's a step-change in how we deliver therapies in this area; a new approach that activates a patient's own immune system which is both more effective and less toxic than standard chemotherapy," said Professor Adele Fielding, Professor of Haematology, University College London. "I am proud that the UK was able to play a key role in the international Phase 2 and 3 studies that led to this approval; and of NICE for evaluating the clinical evidence that is generated by such trials, for their transparent, rational and fair approach to decision making."

The clinical evidence assessed by NICE focused on the Phase 3 TOWER study and Phase 2 MT 103-211 study. Results from the TOWER study showed that median overall survival (OS) was 7.7 months (95 percent CI: 5.6, 9.6) for blinatumomab versus 4.0 months (95 percent CI: 2.9, 5.3) for standard of care chemotherapy (hazard ratio [HR] for death=0.71; p=0.01).^[2]

"Amgen is committed to ensuring patients in the UK get access to effective therapies for hard-to-treat cancers like ALL. Patients with relapsed or refractory ALL have an extremely poor prognosis and we are pleased that NICE has recommended blinatumomab for routine use on the NHS, giving patients and their physicians a novel option to help manage their disease," said Tony Patrikios, Executive Medical Director, Amgen UK and Ireland.

ALL is a rare and rapidly progressing cancer of the blood and bone marrow, with around 760 new cases in the UK a year.^[3] Currently, there is no broadly accepted standard treatment regimen for adult patients with relapsed or refractory ALL beyond chemotherapy.^[4] In adults, approximately 75 percent is B-cell precursor ALL, of which 75-80 percent are Philadelphia-chromosome-negative, and roughly half will be refractory to treatment or experience relapse.^[5]

In England, blinatumomab is available with immediate effect via interim funding arrangements. In Wales, medicines recommended by NICE will be made available within two months of the publication of the Final Appraisal Determination. Subject to NICE issuing positive Final Guidance, interim funding for blinatunomab in England will be available until 90 days after NICE Final Guidance is issued, whereupon funding will switch permanently to baseline commissioning budgets. This guidance is contingent upon Amgen UK providing blinatumomab to the NHS within the terms of an agreed patient access scheme.

Please refer to the Summary of Product Characteristics for full European prescribing information: https://www.medicines.org.uk/emc/medicine/31231

About BLINCYTO^® (blinatumomab)
Blinatumomab is a bispecific CD19-directed CD3 T cell engager (BiTE^®) antibody construct that binds specifically to CD19 expressed on the surface of cells of B-lineage origin and CD3 expressed on the surface of T cells.

In November 2015, BLINCYTO was granted conditional marketing authorisation in the EU for the treatment adults with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL).

▼ Important EU Product Safety Information

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

The adverse reactions described in this section were identified in the Phase 2 MT 103-211 study (N=189).The most serious adverse reactions that may occur during blinatumomab treatment include: infections (31.7%), neurologic events (16.4%), neutropenia/febrile neutropenia (15.3%) cytokine release syndrome (0.5%), and tumor lysis syndrome (0.5%). The most common adverse reactions were: infusion-related reactions (67.2%), infections (63.0%), pyrexia (59.8%), headache (34.4%), febrile neutropenia (28%), peripheral edema (25.9%), nausea (24.3%), hypokalemia (23.8%), constipation (20.6%), anemia (20.1%), cough (18.5%), diarrhea (18.0%), tremor (17.5%), neutropenia (17.5%), abdominal pain (16.9%), insomnia (15.3%), fatigue (15.3%), and chills (15.3%).

About BiTE^® Technology

Bispecific T cell engager (BiTE^®) antibody constructs are a type of immunotherapy being investigated for fighting cancer by helping the body's immune system to detect and target malignant cells. The modified antibodies are designed to engage two different targets simultaneously, thereby juxtaposing T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. BiTE^® antibody constructs help place the T cells within reach of the targeted cell, with the intent of allowing T cells to inject toxins and trigger the cancer cell to die (apoptosis). BiTE^® antibody constructs are currently being investigated for their potential to treat a wide variety of cancers.

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 companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

For more information, visit http://www.amgen.co.uk.

References:

1. NICE Final Appraisal Documentation Blinatumomab for previously treated Philadelphia-chromosome-negative acute lymphoblastic leukemia. Available from: https://www.nice.org.uk/guidance/gid-ta10093/documents/final-appraisal-determination-document (Last Accessed April 2017)

2. Topp M, et al. Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia. N Engl J Med 2017; 376:836-847

3. Cancer Research UK Acute lymphoblastic leukaemia (ALL) incidence. Available from: http://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/leukaemia-all#heading-Zero (Last Accessed April 2017)

4. Davis T, Farag SS. Treating relapsed or refractor Philedelphia chromosome-negative actuve lymphoblastic leukemia : liposome-encapsulated vincristine. Int J Nanomedicine. 2013;8:3479-88

5. Katz AJ, et al. Acute lymphoblastic leukemia: an assessment of international incidence, survival, and disease burden. Cancer Causes Control. 2015 Nov;26(11):1627-42