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New blood cancer drug approved for use in England and Wales

written by

Leukaemia Care, Charity

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The National Institute for Health and Care Excellence (NICE) has today approved panobinostat as a treatment option for NHS patients with myeloma in England and Wales.  

Panobinostat, marketed by Novartis as Farydak®, has been recommended in combination with bortezomib and dexamethasone “for adult patients with relapsed and/or refractory multiple myeloma who have received at least 2 prior regimens, including bortezomib and an immunomodulatory agent”. The decision follows the results of the recent Final Appraisal Determination which, following an appeal period, has now been confirmed.

Myeloma is a cancer which affects the plasma cells, a type of white blood cell which is made in the bone marrow (the “spongy” material found in our bones where blood cells are made). Normally, new plasma cells are produced to replace old, worn-out cells in an orderly and controlled way. They produce antibodies, also called immunoglobulins, to help fight infection. In myeloma, however, these plasma cells become abnormal, and “out of control” and release only one type of antibody – known as paraprotein – which has no useful function and does not fight infection. Myeloma affects multiple places in the body where bone marrow such as the spine, skull, pelvis and the areas around the shoulders and hips. There are around 4,420 new cases diagnosed in England and Wales each year.

The approval has been decided based on evidence put forward by the pharmaceutical company, clinicians and patient organisations (like Leukaemia CARE) in addition to the results of clinical trials. The trials showed that after receiving panobinostat (in combination with bortezomib and dexamethasone) there was an increase in median progression-free survival and also showed an improvement in overall survival. These results are positively received as myeloma is a relapsing-remitting cancer that often becomes resistant to treatment; as such numerous treatment options are often required.

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

“This recent decision by NICE is excellent news for myeloma patients and, more broadly, a positive step in terms of improving access to effective treatment options for people with blood cancer. Myeloma is a complex condition and although treatable, even after successful treatment the remission period is limited. Remission can last months or years but eventually most patients will need further treatment. This demonstrates the need for myeloma patients to have access to additional treatment options.

Furthermore, panobinostat is currently available to NHS patients for the same indication in Scotland (as recommended by the Scottish Medicines Consortium) so it is a welcome change to see that it will be available to patients in England and Wales too. As a patient organisation, we work hard to ensure access to treatment throughout the whole of the UK.”