Kyowa Kirin and GENESIS Pharma announce new partnership for rare disease medicines portfolio in Greece, Cyprus and Malta
Kyowa Kirin and GENESIS Pharma announce new partnership for rare disease medicines portfolio in Greece, Cyprus and Malta
December 20, 2022 – Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin), a Japan-based global specialty pharmaceutical company, and GENESIS Pharma, a leading regional biopharma company operating in the broader region of Southeast Europe, have announced a commercialization agreement for two new orphan-designated medicines in Greece, Cyprus and Malta. Under the terms of the agreement, GENESIS Pharma will commercialize Burosumab for the treatment of X-Linked Hypophosphataemia (XLH) and Tumour-Induced Osteomalacia (TIO) and Mogamulizumab for two subtypes of cutaneous T-cell lymphoma (CTCL), a rare non-Hodgkin lymphoma, upon local market approval.
“At Kyowa Kirin our purpose is to make people smile”, said Abdul Mullick, President of Kyowa Kirin International. “And through partnering with GENESIS Pharma I am really pleased that we are living that very purpose, leveraging both organisations’ expertise and infrastructure to ensure people impacted by these rare conditions across Greece, Cyprus and Malta will have access to the medicines they need.”
Mr. Constantinos Evripides, Managing Director of GENESIS Pharma stated: “Innovation can offer a profound therapeutic benefit to people living with chronic and rare diseases. With a clear vision to support these patients, we always aim to expand our strong network of international partners that invest in promising R&D in challenging therapeutic areas. Kyowa Kirin is a company with a long heritage and commitment to cutting-edge science and we are very happy and proud to work together in order to help patients that live with these rare diseases in the countries that we operate”.
About X-Linked Hypophosphataemia (XLH)
X-Linked Hypophosphataemia (XLH) is a rare metabolic bone disease that impacts children and adults. It is a life-long and progressive disease that typically presents in early childhood, causing lower limb deformities, stunted growth, and bone and joint pain.1 Symptoms such as dental abscesses, osteoarthritis, enthesopathy (issues with the tendons), and hearing loss may also develop during adulthood.2,3,4 As a result of the disease, some adults may require special equipment to improve their mobility.5,6 The physical limitations as well as pain and stiffness caused by XLH can affect people’s ability to work and socialize, their emotional wellbeing, and their capacity for self-care.5
People with XLH have a genetic defect on the X-chromosome, which causes an excessive loss of phosphate through the urine and poor absorption from the gut, resulting in chronically low levels of phosphate in the blood.1,7 Phosphate is a key mineral needed for maintaining the body’s energy levels, muscle function, and the formation of healthy bones and teeth.8,9 XLH is the most common form of hereditary rickets.10 It can sometimes appear in individuals with no family history of the disease but is usually passed down from a parent who carries the defective gene.11
About Tumour-Induced Osteomalacia (TIO)
Tumour-induced osteomalacia (TIO), also known as oncogenic osteomalacia, is an acquired disorder caused by typically small, slow-growing, benign PMTs (phosphaturic mesenchymal tumors).1,2 With fewer than 1,000 cases reported in the medical literature,2 TIO mainly affects adults with a mean onset age of 40 – 45 years.1,3 It is associated with progressive and debilitating musculoskeletal deficits,4,5 ultimately having a detrimental impact on ability to perform daily activities, as well as on physical and social wellbeing.6
TIO is characterised by chronic hypophosphataemia caused by tumour(s) secreting excess fibroblast growth factor 23 (FGF23),1 which can lead to issues such as decreased intestinal absorption of phosphate and compromised vitamin D activation.1,2
The most common signs and symptoms include bone pain, walking difficulty, pathological fractures, height loss and muscle weakness.4 In TIO, muscle weakness and pain severely interfere with physical functioning, including standing up without assistance, walking and ability to work.6 The pain in TIO also severely interferes with mood and moderately interferes with enjoyment of life for those living with it.6
TIO diagnosis is often missed and/or delayed and testing serum phosphate levels is important for diagnosis.1
About cutaneous T-cell lymphoma (CTCL): Mycosis fungoides (MF) and Sézary syndrome (SS)
Mycosis fungoides (MF) and Sézary syndrome (SS) are two subtypes of cutaneous T-cell lymphoma (CTCL), a rare type of non-Hodgkin’s lymphoma that can affect the skin, blood, lymph nodes and internal organs.1 CTCL is associated with disfiguring skin lesions, intractable itching, sleep disturbance and psychosocial problems and has a serious negative effect on quality of life.2
The annual incidence of CTCL in Europe is around 5.2 new cases per 100,000 population3 and it affects around 240 people per million in the region at any one time.4 MF accounts for approximately 60% of all CTCLs,5 is typically indolent and is characterized by skin symptoms including patches or plaques, skin redness and tumours.6 SS is much rarer, accounting for around 5% of CTCLs,7 and is more aggressive8 with high levels of blood involvement by definition.5 It causes very severe itching, total body redness (erythroderma), intense scaling of the skin and frequent hair loss.9
It can typically take between three and six years from CTCL disease onset for a patient to be diagnosed, in some cases it can take decades.5,6 This is sometimes due to the fact that CTCL presents very similarly to other benign skin conditions (e.g. eczema and psoriasis)9; this can mean many patients experience a long, frustrating journey before diagnosis.
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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 a heritage of more than 70 years, the company applies cutting-edge science, including expertise in antibody research and engineering, to address the needs of patients across multiple therapeutic areas such as nephrology, oncology, immunology/allergy and neurology. Across its four regions – Japan, Asia Pacific, North America and EMEA/International – Kyowa Kirin focuses on its purpose, to make people smile, and is united by its shared values of commitment to life, teamwork, innovation and integrity.
You can learn more about the business of Kyowa Kirin at: www.international.kyowa-kirin.com
About GENESIS Pharma
GENESIS Pharma is a regional biopharma company focused on the commercialization of innovative biopharmaceutical products targeting severe and rare diseases in Southeast Europe. Established in 1997, GENESIS Pharma was among the first pharmaceutical companies in Europe to specialize in the marketing, sales and distribution of biopharmaceutical products. GENESIS Pharma maintains a strong portfolio in therapeutic areas with high unmet medical need through long standing strategic alliances with some of the leading global biopharma companies.
For more information, please visit www.genesispharmagroup.com and www.genesispharma.com
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REFERENCES
X-LINKED HYPOPHOSPHATAEMIA
1 Skrinar A, Dvorak-Ewell M, Evins A, et al. The lifelong impact of X-linked hypophosphatemia: Results from a burden of disease survey. J Endocr Soc. 2019;3:1321-1334.
2 Lee JY, Imel EA. The changing face of hypophosphatemic disorders in the FGF-23 era. Pediatr Endocrinol Rev. 2013;10:367-379.
3 Che H, Roux C, Etcheto A, et al. Impaired quality of life in adults with X-linked hypophosphatemia and skeletal symptoms. Eur J Endocrinol. 2016;174:325-333.
4 Ruppe MD. X-Linked Hypophosphatemia. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews®. Seattle (WA): University of Washington, 1993-2018. Available at: https://www.ncbi.nlm.nih.gov/books/NBK83985/. Last accessed: November 2022.
5 Lo SH, Lachmann R, Williams A, et al. Exploring the burden of X-linked hypophosphatemia: a European multi-country qualitative study. Qual Life Res. 2020;29:1883-1893.
6 Theodore-Oklota C, Bonner N, Spencer H, et al. Qualitative research to explore the patient experience of X-linked hypophosphatemia and evaluate the suitability of the BPI-SF and WOMAC® as clinical trial end points. Value Health. 2018;21:973-983.
7 Beck-Nielsen SS, Mughal Z, Haffner D, et al. FGF23 and its role in X-linked hypophosphatemia-related morbidity. Orphanet J Rare Dis. 2019;14:58.
8 Pesta D, Tsirigotis DN, Befroy DE, et al. Hypophosphatemia promotes lower rates of muscle ATP synthesis. The FAESB Journal. 2016;39:3378-3387.
9 Unnanuntana A, Rebolledo BJ, Khair MM, et al. Diseases affecting bone quality: beyond osteoporosis. Clin Orthop Relat Res. 2011;469:2194-2206.
10 Carpenter TO, Imel EA, Holm IA, et al. A clinician's guide to X-linked hypophosphatemia. J Bone Miner Res. 2011;26:1381-8.
11 National Center for Advancing Translational Sciences. X-linked hypophosphatemia. Available at: https://rarediseases.info.nih.gov/diseases/12943/x-linked-hypophosphatemia. Last updated: February 2018. Last accessed: November 2022
TUMOUR-INDUCED OSTEOMALACIA
1 Brandi ML, et al. Challenges in the management of tumor-induced osteomalacia (TIO). Bone. 2021;152:116064.
2 Florenzano P, et al. Tumor-Induced Osteomalacia. Calcified Tissue International. 2021;108:128-42.
3 Dahir K, et al. Diagnosis and Management of Tumor-Induced Osteomalacia: Perspectives from Clinical Experience. Journal of the Endocrine Society. 2021;5:1-12.
4 Feng J, et al. The diagnostic dilemma of tumor induced osteomalacia: a retrospective analysis of 144 cases. Endocrine Journal. 2017;64:675-83.
5 Minisola S, et al. Tumour-induced osteomalacia. Nature Reviews Disease Primers. 2017 ;3 :17044.
6 Jerkovich F, et al. Burden of Disease in Patients with Tumor-Induced Osteomalacia. JBMR Plus. 2020;5:e10436.
MYCOSIS FUNGOIDES AND SEZARY SYNDROME
1 Olsen E, Vonderheid E, Pimpinelli N, et al. Blood. 2007;110(6):1713-22
2 EHA 2021 abstract. Available at: https://library.ehaweb.org/eha/2021/eha2021-virtual-congress/324119/mark.bishton.health-related.quality.of.life.effect.of.mogamulizumab.by.patient.html?f=listing%3D0%2Abrowseby%3D8%2Asortby%3D1%2Asearch%3Dpb1439 Accessed November 2022
3 Orphanet. Prevalence and incidence of rare diseases: Bibliographic data. January 2021. Available at: https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf.
Accessed: November 2022.
4 Willemze R, et al. Blood. 2019;133(16):1703–1714
5 Wilcox R. Am J Hematol. 2016;91(1):151-165.
6 Scarisbrick J, et al. Br J Dermatol. 2019;181(2):350–357.
7 Girardi M, Heald PW, Wilson LD. N Engl Jnl Med. 2004; 350(19): 1978-1988
8 Lymphoma Coalition. Cutaneous lymphoma – a patient’s guide. Available at:
https://lymphomacoalition.org/wp-content/uploads/Cutaneous_lymphoma_-_patients_guide_-.pdf.
Accessed November 2022
9 Demierre M, et al. Cancer. 2006;107(10):2504–2511
