The Scottish Medicines Consortium (SMC) Accepts Kyowa Kirin’s POTELIGEO® (mogamulizumab) for the Treatment of Those Living With Certain Ultra-rare Blood Cancers

Adults with two forms of ultra-rare non-Hodgkin lymphoma living in Scotland will now have access to an innovative systemic treatment following SMC’s decision to accept mogamulizumab for restricted use by NHS Scotland.

GALASHIELS, Scotland–(BUSINESS WIRE)–The Scottish Medicines Consortium (SMC) 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.¹

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

About Kyowa Kirin

Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company with over 70-year heritage, we apply cutting-edge science including an expertise in antibody research and engineering, to address the needs of patients and society across multiple therapeutic areas including Nephrology, Oncology, Immunology/Allergy and Neurology. Across our four regions – Japan, Asia Pacific, North America and EMEA/International – we focus on our purpose, to make people smile, and are united by our shared values of commitment to life, teamwork/Wa, innovation, and integrity. You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com.

KK/UK/POT/0015

Date of Preparation: June 2021

References

¹ Scottish Medicines Consortium. Advice on new medicines – mogamulizumab 4mg/mL concentrate for solution with infusion (POTELIGEO®). SMC2336. Available from https://www.scottishmedicines.org.uk/medicines-advice/mogamulizumab-poteligeo-full-smc2336/. Last Accessed: June 2021.

² European Medicines Agency (EMA). POTELIGEO 4mg/mL, concentrate for solution for infusion – product information. Available from https://www.medicines.org.uk/emc/product/11174. Last Accessed: June 2021.

³ Gilson, D, et al. British Association of Dermatologists and UK Cutaneous Lymphoma Group Guidelines for the management of primary cutaneous lymphoma. British Journal of Dermatology. 2019. 180. pp.496-526

⁴ National Organization for Rare Disorders: Cutaneous T-Cell Lymphomas. Available from: https://rarediseases.org/rare-diseases/cutaneous-t-cell-lymphomas/. Last Accessed: June 2021.

⁵ Williams et al (2020) – Health state utilities associated with caring for an individual with CTCL. Journal of Medical Economics. 2020; 23(10):1142-1150.

⁶ Ferenczi K, Fuhlbrigge RC, Pinkus J, et al. Increased CCR4 expression in cutaneous T cell lymphoma. J Invest Dermatol. 2002;119:1405-10.

⁷ Yoshie O, et al. Frequent Expression of CCR4 in Adult T-Cell Leukemia and Human T-cell Leukemia Virus Type 1-transformed T cells. Blood. 2002;99(5):1505-11.

⁸ Ishida T, et al. Clinical Significance of CCR4 Expression in Adult T-cell Leukemia/Lymphoma: Its Close Association With Skin Involvement and Unfavorable Outcome. Clin Cancer Res. 2003;9:3625-34.

⁹ Duvic M, et al. Mogamulizumab for the treatment of cutaneous T-cell lymphoma: recent advances and clinical potential. Ther Adv Hematol. 2016;7(3):171-174.

¹⁰ Kim YH, Bagot M, Pinter-Brown L, et al. Mogamulizumab versus vorinostat in previously treated cutaneous T-cell lymphoma (MAVORIC): an international, open-label, randomised, controlled phase 3 trial. Lancet Oncol. 2018;19(9):1192-1204.

¹¹ Cutaneous Lymphoma Foundation, Lymphoma Action and Lymphoma Coalition Europe. Cutaneous lymphoma – a patient’s guide. 2019. Available from: https://lymphoma-action.org.uk/sites/default/files/media/documents/2019-06/Cutaneous%20lymphoma%20-%20patient%26%23039%3Bs%20guide%20-%20English%20language%20source%20document%20-%20final%20version%20for%20publication%20-%20April%202019.pd Last accessed: June 2021.

¹² Mariani M, Lang R, Binda E, et al. Dominance of CCL22 over CCL17 in induction of chemokine receptor CCR4 desensitization and internalization on human Th2 cells. Eur J Immunol. 2004;34(1):231-240.

¹³ Wilcox RA. Cutaneous T-cell lymphoma: 2016 update on diagnosis, risk-stratification, and management. Am J Hematol. 2016;91(1):151-65.

¹⁴ Ni X, Jorgensen JL, Goswami M, et al. Reduction of regulatory T cells by Mogamulizumab, a defucosylated anti-CC chemokine receptor 4 antibody, in patients with aggressive/refractory mycosis fungoides and Sézary syndrome. Clin Cancer Res. 2014; 21(2):274-85.

¹⁵ Kakinuma T, Sugaya M, Nakamura K, et al. Hymus and activation-regulated chemokine (TARC/CCL17) in mycosis fungoides: serum TARC levels reflect the disease activity of mycosis fungoides. J Am Acad Dermatol. 2003;48(1):23-30.

¹⁶ Girardi M, Heald PW, Wilson LD. The Pathogenesis of Mycosis Fungoides. NEJM. 2004;350(19):1978-88.

¹⁷ Olsen E, Vonderheid E, Pimpinelli N, et al. Revisions to the staging and classification of mycosis fungoides and Sezary syndrome: a proposal of the International Society for Cutaneous Lymphomas (ISCL) and the cutaneous lymphoma task force of the European Organization of Research and Treatment of Cancer (EORTC). Blood. 2007;110(6):1713-22.

¹⁸ Scarisbrick JJ, Prince M, Vermeer MH, et al. Cutaneous Lymphoma International Consortium Study of Outcome in Advanced Stages of Mycosis Fungoides and Sézary Syndrome: Effect of Specific Prognostic Markers on Survival and Development of a Prognostic Model. J Clin Oncol. 2015;33(32):3766-3773.

¹⁹ Willemze R, Hodak E, Zinzani PL et al. Primary cutaneous lymphomas: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2018;29(4):1-29.

²⁰ Kim EJ, Hess S, Richardson SK, et al. Immunopathogenesis and therapy of cutaneous T cell lymphoma. J Clin Invest. 2005;115(4):798-812.

²¹ Scarisbrick JJ, Whittaker, S, Evans, AV, et al. Prognostic significance of tumor burden in the blood of patients with erythrodermic primary cutaneous T-cell lymphoma. Blood. 2001;97(3):624-30.

²² CL Foundation: A Patient’s Guide. Available from: https://www.clfoundation.org/sites/default/files/2018-04/a_patients_guide.pdf. Last Accessed: May 2021.

²³ Agar N, et al. Survival Outcomes and Prognostic Factors in Mycosis Fungoides/Sezary Syndrome: Validation of the Revised International Society for Cutaneous Lymphomas/European Organisation for Research and Treatment of Cancer Staging Proposal. J Clin Ocol. 2010;28(31):4730-4739.

²⁴ Krejsgaard T, Lindahl LM, Mongan NP, et al. Malignant inflammation in cutaneous T-cell lymphoma—a hostile takeover. Semin Immunopathol. 2017;39(3):269–282.

²⁵ Orphanet: Mycosis Fungoides. Available from: https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Lng=GB&Expert=2584. Last Accessed: May 2021.

²⁶ Orphanet: Sézary syndrome. Available from: https://www.orpha.net/consor/cgi-bin/OC_Exp.php?Expert=3162. Last Accessed: May 2021.

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Email: [email protected]