On June 7, 2021 The Scottish Medicines Consortium (SMC) reported that has accepted POTELIGEO (mogamulizumab) for restricted use by NHS Scotland for the treatment of adults with advanced mycosis fungoides (MF) or Sézary syndrome (SS) (stage ≥IIB MF and all SS) following at least one prior systemic therapy, who are clinically ineligible for or refractory to treatment with brentuximab vedotin (Press release, Kyowa Hakko Kirin, JUN 7, 2021, View Source [SID1234583690]).1

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The ultra-rare blood cancers mycosis fungoides and Sézary syndrome are two subtypes of cutaneous T-cell lymphoma (CTCL).1,2 The medicine was accepted following consideration through the SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines for end of life and/or ultra-rare conditions.

Richard Johnson, Northern Cluster General Manager, responsible for the UK at Kyowa Kirin, commented: "We are delighted that eligible adults with MF and SS can now access mogamulizumab through NHS Scotland. Today’s advice from the SMC marks an important milestone and is testament to the hard work, collaboration and commitment of the wider CTCL community and the SMC, to ensure those living with these ultra-rare blood cancers have an innovative treatment option in an area of high unmet need."

Dr Pam McKay, Consultant Haematologist and Honorary Clinical Associate Professor at The Beatson – West of Scotland Cancer Centre added: "This is a welcome decision from the SMC which means another treatment option will be available for those living with MF and SS. It has been well documented that those who are at the advanced stages of CTCL have a poor prognosis3 and very few licensed, systemic and well tolerated therapeutic options available to them. Furthermore, those living with CTCL also have a substantially reduced quality of life as the condition causes an immense impact upon their day-to-day functions as well as on the lives of their carers and loved ones. The use of an effective therapy such as mogamulizumab, will be greatly welcomed by adults with MF and SS and clinicians in Scotland, who now have an additional option for those currently living with the effects of this debilitating disease."

MF and SS are two forms of CTCL, which is a serious and potentially life-threatening form of cancer.4 Additionally, there is a significant impact on quality of life for those caring for an individual living with CTCL.5 CTCL is treatable but not curable and there is a clear unmet need for new treatment options.

Ropinder Gill, Chief Executive at Lymphoma Action commented: "This is a hugely significant development for those living in Scotland with these rare haematological cancers. Mogamulizumab offers people with MF and SS a much-needed new treatment option for a disease where, to date, therapies have been limited. Lymphoma Action welcomes this decision by the SMC who have recognized that people with MF and SS should have access to the full range of treatment options."

This decision comes after the National Institute for Health and Care Excellence (NICE) recommended not to reimburse mogamulizumab in England and Wales for the treatment of adult patients with certain types of CTCL, namely MF/SS who have received at least one prior systemic therapy.

Kyowa Kirin lodged an appeal against NICE’s recommendation on 18 March 2021 and appeals were also lodged by Lymphoma Action & Leukaemia Care (joint submission) and the UK Cutaneous Lymphoma Group (UKCLG). The NICE appeal panel convened on 10 May 2021 to hear oral representations from the appellants and a final decision regarding the appeal is expected in July 2021. Kyowa Kirin remains committed to finding a solution for people living with SS/MF to have access to POTELIGEO in England and Wales and will continue to work with NICE and NHS England to find a resolution.

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;6,7,8 once mogamulizumab binds to CCR4, it increases attraction of immune cells from the immune system to destroy the cancerous cells.9

Mogamulizumab has been shown to offer benefits to many patients with MF and SS.10 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).10 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.10 Levels of adverse events were similar between the two treatment groups.10 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).10

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

MF and SS are characterised by localisation of cancerous white blood cells called T lymphocytes (T cells), to the skin.11,12 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.6,7,8 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 6,13,14,15,16 which can resemble psoriasis or eczema.11

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.17 All four areas of the body are used to assess disease stage,18,19 and clinically significant involvement of the blood, particularly in more advanced disease, is linked with increased morbidity and an overall reduction in patient survival.18,20,21

CTCL can take, on average, between 2 and 7 years for individuals to receive a confirmed diagnosis.22 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.23 Whilst most individuals that present with early stage disease do not progress to a more severe stage,24 patients with advanced disease have significantly poorer outcomes with only around half of patients (52%) surviving for just 5 years.18

CTCL is a ultra-rare disease that affects 0.7 per 100,000 patients across the UK.3 The annual incidence of MF in Europe is estimated to be between 1 in 110,000 to 1 in 350,000.25 The annual incidence of SS is 1 in 10,000,000.26 Together they represent approximately 65% of all cases of CTCL.17

*1 Vorinostat is a USA FDA-licensed existing treatment for MF and SS and is currently unlicensed in the EU