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New blood cancer drugs available to patients in the UK and EU

written by

Leukaemia Care, Charity

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European access to blood cancer treatments

The European Medicines Agency (EMA) is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union (EU), in order to enable European patients’ access to innovative drugs, whilst safeguarding public health. The EMA has recently approved two new blood cancer treatments.


Blinatumomab, marketed by Amgen as Blincyto®, has recently been approved by the EMA as a treatment in Europe for adults with Philadelphia chromosome-negative acute lymphoblastic leukaemia.

Acute lymphoblastic leukaemia (ALL) is a rapidly progressing type of leukaemia that is caused by the overproduction of immature B lymphocytes (a type of white blood cell that make antibodies to help fight infection) which is produced in the bone marrow (the spongy tissue found inside the bones). The overproduction of these cells means that the body is unable to fight infection. On average there are around 900 cases of Philadelphia chromosome-negative ALL in Europe each year, with an estimated 88 cases in the UK.

The EMA has specifically recommended the drug “for the treatment of adults with Philadelphia chromosome negative relapsed or refractory B-precursor acute lymphoblastic leukaemia”. The approval follows the results of Phase II trials which concluded that 42.9% of patients achieved complete remission or a partial haematological response after being treated with blinatumomab.  This is a welcome result as the outlook for adult patients with relapsed or refractory ALL is particularly poor.

This sanction allows for the marketing authorisation of blinatumomab throughout Europe for the recommended indication however, before it can be prescribed to NHS patients in the UK, it will require appraisal by and need approval from the relevant UK health technology appraisal (HTA) bodies.


Carfilzomib, which is marketed by Amgen as Kyprolis ®, has also been approved by the EMA in combination with lenalidomide and dexamethasone for multiple myeloma patients who have received at least one prior therapy.

 Multiple myeloma is a cancer which affects the plasma cells, a type of white blood cell found in the bone marrow. Plasma cells produce antibodies called ‘immunoglobulins’ to help fight infections. Normally new plasma cells are created to replace old cells, but in patients with myeloma abnormal amounts of plasma cells are produced which only produce one type of antibody called ‘paraprotein’ which has no useful function and cannot fight infection effectively. Treatment can be very effective at controlling symptoms and stopping the development of the disease but it is rarely curable. There are around 39,000 European patients diagnosed with myeloma each year, with 4,420 new cases of these in England and Wales.

The decision by the EMA is based on a clinical study that showed that carfilzomib improved progression-free survival and patients taking the drug lived for an average of 26.3 months without disease progression.  Importantly, the EMA noted that there was an unmet need for patients with multiple myeloma who no longer responded to existing therapies.

Again, before this treatment is available to NHS patients in the UK it will need to be appraised and approved by the relevant UK health technology appraisal body.

UK access to blood cancer treatment

Patients in England and Wales could soon get access to two new blood cancer treatments. The National Institute for Health and Care Excellence (NICE) appraises drugs and health technologies and develops guidance on the use of new and existing medicines. It decides what treatments should be available to patients via the NHS in England and Wales.

Recently, NICE has published positive Final Appraisal Determinations (FAD) for two blood cancer drugs. This means that the drugs have been found to be an effective, cost effective treatment option (although subject to appeal) and should soon be available to NHS patients in England and Wales.


Firstly, NICE has published an FAD provisionally recommending “bortezomib in combination with rituximab, cyclophosphamide, doxorubicin and prednisone (VR-CAP) in adult patients with previously untreated mantle cell lymphoma… for whom haematopoietic stem cell transplantation is unsuitable”.

Mantle cell lymphoma (MCL) is a rare, high grade form of non-Hodgkin lymphoma (NHL). It develops quickly and, if not treated, grows quickly. Lymphoma affects types of white blood cells called lymphocytes – “mantle cell” describes the type of lymphocyte that is affected. It occurs when some of the white blood cells divide in an abnormal way and do not die as and when they should. These abnormal cells can collect in the lymph nodes which then enlarge as tumours form. The number of new MCL cases in the UK each year is about 510.

The NICE decision to approve bortezomib (already marketed by Janssen for the treatment of myeloma as Velcade®) follows a clinical study which showed that when treated with bortezomib (in combination with VR-CAP) progression-free survival was statistically significantly better for adults with previously untreated mantle cell lymphoma (who were unsuitable for stem cell transplant) compared to its comparator treatment option of rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP).


NICE have also given an initial nod of approval for panobinostat in combination with bortezomib and dexamethasone for patients with myeloma. The NICE FAD recommends the therapy, marketed by Novartis as Farydak ®, for the treatment of “adult patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent.” The decision is subject to appeal but if approved could mean that myeloma patients in England and Wales could have access to a new, effective treatment option.

As previously mentioned, myeloma is a cancer which affects the plasma cells, a type of white blood cell found in the bone marrow and there are around 4,420 new cases diagnosed in England and Wales each year.

This initial approval has been decided based on a clinical trial which showed that, after receiving panobinostat in combination with bortezomib and dexamethasone, the median progression-free survival extension was “7.8 months, representing a 53% reduction in the risk of progression”. It also showed an improvement in overall survival, a welcomed response in a “complex blood cancer that often becomes resistant to treatment”.

As indicated, both of these decisions are subject to appeal but if the final decision goes ahead, then it means improved access to blood cancer drugs for NHS patients in England and Wales.

Zack Pemberton-Whiteley, Head of Campaigns and Advocacy at Leukaemia CARE has commented:

“The recent movements of both the EMA and NICE in terms of enabling access to blood cancer drugs is welcomed by Leukaemia CARE. We actively campaign for increased access to effective blood cancer treatments that will both increase life expectancy and patients’ quality of life. We hope that the treatments that have been approved by the EMA will be available in UK in the future and that the initial NICE FAD decisions are upheld. If they are, this will be good news for blood cancer patients in England and Wales.”