(PharmaNewsWire.Com, December 17, 2019 ) Hunter Syndrome Treatment Market The global Hunter Syndrome Treatment Market is expected to grow at a CAGR of just over 7 % during the forecast period (2019-2026).
Hunter syndrome is also known as mucopolysaccharidosis type II, and it affects several parts of the body. It is an inherited genetic disorder (an X-linked recessive disorder) caused by the missing or malfunctioning iduronate-2-sulfatase enzyme and can be severe or mild. Patients are suffering from a severe form of the disease experience a rapid decline in intellectual function as it progresses. Patients start losing basic functional skills between the ages of 6 and 8. The normal life expectancy of these people reduces to 10 to 20 years. Mild type condition also reduces the life span, but the individuals live into adulthood without getting their intelligence effected. Major causes of death in individuals with Hunter syndrome is airway obstruction and heart diseases. Diagnostic tests such as iduronate-2-sulfatase (I2S) enzyme activity test, genetic testing, and urine test are performed to diagnose the disease.
Hunter syndrome has no permanent cure; hence, the treatment mainly focuses on the management of indications and difficulties associated with disease progression. Hunter syndrome’s treatment regime is designed based on the severity of symptoms and varies from patient-to-patient. According to the National Institute of Health (NIH) in 2018, Hunter syndrome affected around one per 160,000 males.
Market Dynamics The market is driven by a rising patient population of Hunter syndrome, potential approval of pipeline drugs globally, increasing healthcare expenses, fee reductions and grants, and favorable regulations.
Success for the drugs in pipeline with faster market access and favorable regulations for their approval are needed to cope up with the fast-growing prevalence of the disease. To increase patient benefits, novel therapeutics such as gene therapy and RNA therapeutics are gaining popularity. In 2018, the National Institute of Neurological Disorders and Stroke (NINDS) funded research for the termination of the material buildup in brain cells through a virus delivered gene therapy showed improvement in memory and learning.
The high cost of therapy and lack of awareness can hamper the growth of the market in the forecast period.
Market Segmentation The global Hunter syndrome treatment market can be segmented by treatment type as Enzyme Replacement Therapy (ERT), Hematopoietic Stem Cell Transplant (HSCT) and others and by end-users – hospitals, clinics, specialty care unit and others.
Based on the treatment type, ERTs are expected to dominate the market in the period of forecast. ERT uses an IV to replace a deficient or missing enzyme in the body. ERT does not cure the disease but slows its progression by increasing the number of missing enzymes in the body. Elaprase (idursulfase) is a long-term ERT for patients with a confirmed diagnosis of MPS II and has been approved for use in the US, Canada, and many countries in Europe. Elaprase is developed and manufactured by Shire Pharmaceuticals and is given as weekly infusions. Strong sales of Elaprase and potential worldwide approval for the treatment of Hunter syndrome are some of the factors responsible for the rapid growth of ERT. Shire’s Elaprase (idursulfase) is the single primary drug for the treatment of mucopolysaccharidosis type worldwide, with GC Pharma’s Hunters (idursulfase beta) being approved only in South Korea currently. According to NIH, around 2,000 patients globally are candidates for enzyme replacement therapy.
Market Geographical Analysis Geographically, the global hunter syndrome treatment market is divided into North America, Europe, South America, Asia-Pacific, and the Middle East and Africa. North America is expected to account for the predominant market share in the period of forecast due to favorable government regulations for orphan drug development, improving awareness on diagnosis and treatment of rare diseases, increased funding for research activities and improved healthcare facilities. The US government provides health insurance coverage and reimbursement of expensive drugs such as Elaprase for the treatment of Hunter syndrome Asia Pacific region is expected to exhibit the highest CAGR for forecast due to the large target population in emerging countries such as India and China, improvement in healthcare infrastructure and significant opportunities for biopharmaceutical companies to penetrate the region.
Market Competitive Analysis Some of the major key players in the market are Takeda Pharmaceutical Company Limited, GC Pharma, JCR Pharmaceuticals Co Ltd., RegenxBio Inc., Sangamo Therapeutics, Inc., ArmaGen Inc., Inventiva S.A., Denali Therapeutics Inc., Bioasis Technologies Inc., BioMarin Pharmaceutical, Inc., and Esteve.
Key players in the market are engaged in the development of novel technologies, strategic planning, and alliances to achieve extensive growth, acquire a leading position in the market, and increase geographical footprints.
In January 2019, Takeda Pharmaceutical Company Limited acquired Shire plc expanding its geographic footprint and leading position in Japan and the U.S.
In November 2018, REGENXBIO Inc. announced that the first subject in Phase I/II study of RGX-121 has been successful for the treatment of Mucopolysaccharidosis Type II.
In June 2018, JCR Pharmaceuticals Co., Ltd. announced that Pharmaceutical and Medical Devices Agency (PMDA), completed the review of the Phase 3 clinical trial plan for JR-141 in Japan.
In May 2018, FDA granted fast track designation for RGX-121 that is a novel, one-time investigational treatment for Mucopolysaccharidosis Type II, that is designed to deliver the human iduronate 2-sulfatase (I2S) gene directly to the CNS using the NAV AAV9 vector.
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