On November 11, 2021 Kyowa Hakko Kirin reported that The National Institute for Health and Care Excellence (NICE) has published positive guidance, in the form of a Final Appraisal Determination (FAD), confirming that POTELIGEO (mogamulizumab) is recommended as a treatment option for adults in England and Wales with the ultra-rare blood cancers mycosis fungoides (MF) and Sézary syndrome (SS), two forms of cutaneous T-cell lymphoma (CTCL) (Press release, Kyowa Hakko Kirin, NOV 11, 2021, View Source [SID1234595297]). The treatment is approved for use after two prior systemic treatments for adults living with MF and after one prior systemic therapy for adults living with SS.1

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The FAD comes following a successful appeal lodged by Kyowa Kirin, Lymphoma Action, Leukaemia Care, and the UK Cutaneous Lymphoma Group (UKCLG) in response to a previous appraisal by NICE, published on 4 March 2021, that concluded POTELIGEO would not be made available on the NHS in England and Wales.2

All parties involved in lodging the appeal welcome the new, positive decision by NICE as it represents a distinctive step forward in addressing the high unmet clinical needs of those living with MF and SS. This decision also marks another milestone in the availability of POTELIGEO across the United Kingdom, following the acceptance of POTELIGEO by the Scottish Medicines Consortium (SMC) for adults living with MF and SS in Scotland on 7 June 20213.

Richard Johnson, Northern Cluster General Manager, responsible for the UK at Kyowa Kirin, said: "This is a momentous day for those living with MF and SS in the UK. By issuing positive guidance for POTELIGEO, NICE has helped ensure that those with MF and SS who have few systemic treatment options available to them are now able to access an innovative therapy. This decision is also testament to the dedication, commitment and long-term efforts of the broader CTCL community in advocating for a wider range of treatment options in the UK for those living with conditions like MF and SS."

Ropinder Gill, Chief Executive at Lymphoma Action, commented: "We’re really grateful and delighted with this decision. Mogamulizumab can have a life changing impact for those people affected by cutaneous T-cell lymphoma, a form of the rare condition skin lymphoma. They have limited treatment options – this is a great outcome for them. We were privileged to be part of the process alongside others, and to represent the patient voice with our patient advocate Stan."

Prof Julia Scarisbrick, consultant dermatologist and head of the Cutaneous Lymphoma Service within the Rare Diseases Centre at University Hospital Birmingham, added: "Adults living with CTCL have significant unmet clinical need and a lack of treatment options available to them. There has been a real need to address this for a long time and particularly so for those at the advanced stages of the disease, who have a poorer prognosis and impaired health related quality of life.4 With NICE’s decision, adults with MF and SS across the whole UK now have an effective, well tolerated therapy shown to improve quality of life available to them that can provide a welcome alternative to those who need it."

About POTELIGEO (mogamulizumab)

Mogamulizumab is a first-in-class humanised monoclonal antibody (mAb) directed against CC chemokine receptor 4 (CCR4), a protein consistently expressed on cancerous cells seen in both MF and SS;5,6,7 once mogamulizumab binds to CCR4, it increases attraction of immune cells from the immune system to destroy the cancerous cells.8

Mogamulizumab has been shown to offer benefits to many patients with MF and SS.9 The MAVORIC trial compared the efficacy of mogamulizumab with vorinostat in previously treated people with relapsed or refractory mycosis fungoides or Sézary syndrome, two types of Cutaneous T-cell lymphoma (CTCL).9 Patients taking mogamulizumab experienced control over their disease for more than twice as long as those taking the comparator treatment, vorinostat*1 (7.7 months vs 3.1 months of median progression free survival), the primary endpoint of the trial.9 Levels of adverse events were similar between the two treatment groups.9 The MAVORIC trial is the largest in CTCL; it enrolled a total of 372 patients across 61 sites in 11 countries (of which 16 sites were in Europe, including three in England).9

About Mycosis Fungoides (MF) and Sézary Syndrome (SS)

MF and SS are two forms of CTCL,10 which is a serious and potentially life-threatening form of cancer.11 Additionally, there is a significant impact on quality of life for those caring for an individual living with CTCL.12 CTCL is treatable but not curable and there has been a clear unmet need for new treatment options.

MF and SS are characterised by localisation of cancerous white blood cells called T lymphocytes (T cells), to the skin.13,14 These cancerous T cells consistently express a protein called CC-chemokine receptor 4 (CCR4), which enables them to move from the blood to the skin.5,6,7 When these cancerous T cells move to the skin, they can create a localised inflammatory immune skin response, commonly resulting in visible skin symptoms of red patches or plaques 5,15,16,17,18 which can resemble psoriasis or eczema.13

MF and SS can affect the skin, blood, lymph nodes (part of the body’s immune system which is spread throughout the body) and internal organs.19 All four areas of the body are used to assess disease stage20,21 and clinically significant involvement of the blood, particularly in more advanced disease, is linked with increased morbidity and an overall reduction in patient survival.20,22,23

CTCL can take, on average, between 2 and 7 years for individuals to receive a confirmed diagnosis.24 It is critical for doctors to consider CTCL as an early differential diagnosis as the patient’s prognosis can be affected if the disease progresses to later stages.25 Whilst most individuals that present with early stage disease do not progress to a more severe stage,26 patients with advanced disease have significantly poorer outcomes with only around half of patients (52%) surviving for just 5 years.20

CTCL is an ultra-rare disease that affects 0.7 per 100,000 patients across the UK.4 The annual incidence of MF in Europe is estimated to be between 1 in 110,000 to 1 in 350,000.27 The annual incidence of SS is 1 in 10,000,000.28 Together they represent approximately 65% of all cases of CTCL.19