The “Hunter Syndrome – Market Insights, Epidemiology and Market Forecast – 2030” drug pipelines has been added to ResearchAndMarkets.com’s offering.
This report delivers an in-depth understanding of the Hunter Syndrome, historical and forecasted epidemiology as well as the Hunter Syndrome market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
The Hunter Syndrome market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Hunter Syndrome market size from 2017 to 2030. The report also covers current Hunter Syndrome treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
Hunter Syndrome Diagnosis
The diagnosis of Hunter syndrome is established in a male by identifying the deficient iduronate 2-sulfatase (I2S) enzyme activity in white cells, fibroblasts, or plasma in the presence of normal activity of at least one other sulfatase. Detection of a hemizygous pathogenic variant in IDS confirms the diagnosis in a male with an unusual phenotype or a phenotype that does not match the results of GAG testing. The diagnosis of this indication is usually established in a female with suggestive clinical features by identification of a heterozygous IDS pathogenic variant on molecular genetic testing.
Although the disease is almost exclusively reported in males, rare cases in females also do occur. The diagnosis of MPS II is usually established in a female patient with suggestive clinical features, such as the identification of a heterozygous IDS pathogenic variant on molecular genetic testing.
Molecular genetic testing approaches can include a combination of gene-targeted testing (single-gene testing, multigene panel) and comprehensive genomic testing (exome sequencing, genome sequencing) depending on the phenotype. Gene-targeted testing requires that the clinician determine which gene(s) are likely involved, whereas genomic testing does not.
Hunter Syndrome Treatment
Even with the introduction of ERT, patients with MPS II still require supportive symptomatic treatment from a wide range of specialists. A comprehensive initial assessment of each patient at diagnosis should, therefore, be undertaken, and should be followed by regular reviews. Supportive management and the anticipation of possible complications can greatly improve the quality of life of affected individuals and their families. Family members should be offered genetic counselling, and contact with other affected families, patients, and support groups.
It is now a decade since ERT with intravenous idursulfase (Elaprase), a recombinant form of human iduronate 2-sulfatase, has been approved in the United States and the European Union at a weekly dose of 0.5 mg/kg for the treatment of MPS II. The approval was mainly based on the results from a first trial on individuals with the slowly progressive form of the disease. In the following year several other studies were undertaken to investigate clinical safety and efficacy of ERT; these clearly showed that idursulfase has positive effects on functional capacity (distance walked in six minutes and forced vital capacity), liver and spleen volumes, and urine GAGs excretion. Recently, a 3.5-year independent study determined that long-term use of ERT is similarly effective in young (age 1.6-12 years at the start of ERT) and older individuals (age 12-27 years at the start of ERT). In addition, two recent studies have confirmed ERT efficacy in improving somatic signs and symptoms of the disease in all individuals, including infants younger than age 1 year and individuals with the early progressive MPS II phenotype.
Pretreatment with anti-inflammatory drugs or antihistamines, as is often done for ERT in other conditions, is not suggested on the label for Elaprase; however, if mild or moderate infusion reactions (e.g., dyspnea, urticaria, or systolic blood pressure changes of 20 mm Hg) cannot be ameliorated by slowing the infusion rate, the addition of treatment one hour before infusion with diphenhydramine and acetaminophen (or ibuprofen) to the regimen usually resolves the problem. Pretreatment can typically be discontinued after 6-10 weeks.
Hematopoietic stem cell transplantation (HSCT) using umbilical cord blood or bone marrow is a potential way of providing sufficient enzyme activity to slow or stop the progression of the disease, however, the use of HSCT is controversial because of the associated high risk of morbidity and mortality. The use of HSCT has been controversial because of limited information regarding the long-term outcomes and the associated high risk of morbidity and mortality.
Scope of the Report
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