Global Hunter Syndrome Treatment Market - 2022-2029

Market Overview

Hunter Syndrome Treatment Market is estimated to reach at high CAGR of 7% during the forecast period (2022-2029).

Hunter Syndrome is also known as Mucopolysaccharidosis type II (MPS II). It is a rare genetic disorder in which large sugar molecules called glycosaminoglycans (or GAGs or mucopolysaccharides) build up in body tissues. It is a form of lysosomal storage disease. If Hunter Syndrome affects the brain, symptoms often appear between 18 months and four years. They begin two years later when the condition is milder. Various treatments are available for Hunter Syndrome, like enzyme therapy, stem cell transplant, and gene therapy.

Market Dynamics

The hunter syndrome treatment is driven by innovative novel therapies in clinical trials and increasing R&D activities to develop treatment.

Innovative novel therapies in clinical trials are expected to drive market growth.

The introduction of innovative treatments like gene therapy and RNA therapeutics is in great demand. Enzyme replacement therapy (ERT) with recombinant human I2S (idursulfase) is available for patients with Hunter syndrome. The U.S. Food and Drug Administration and the European Medicines Agency approved idursulfase for treating patients with Hunter syndrome based on a pivotal phase 2/3 randomized, double-blind, placebo-controlled clinical trial in 96 patients with Hunter syndrome aged 5-31 years. Stem Cell Transplant- In this, healthy blood cells are infused into your child's body with the hope that the new cells will create the missing or defective enzyme. Gene Therapy-Replacing the chromosome responsible for producing the missing enzyme could theoretically cure Hunter syndrome, but much research needs to be done. However, research is still on for all these treatments.

The high cost of the treatment and lack of awareness among people are expected to hamper the market growth.

Though the treatment is rare, the treatment for the disorder is only available in U.S. and Europe and is very expensive, and there is no proper diagnosis. Many people are unaware of it. So, governments should create awareness about the disease and its treatment. The Lysosomal Storage Disorders Support Society (LSDSS), a non-profit organization working for several lysosomal disorders, said about 180 children with Hunter Syndrome are registered in India.

Industry Analysis

The hunter syndrome treatment market provides in-depth analysis of the market based on various industry factors such as epidemiology, unmet needs, pricing analysis, supply chain analysis, regulatory analysis etc.

COVID-19 Impact Analysis

The emergence of COVID-19 has impacted the whole world in some or another way. The health crisis has brought an unprecedented impact on businesses across industries. It has resulted in a decrease in public mobility, as well as impacted the diagnostic industries. Several diagnostic procedures and therapies were put on hold to avoid the spread of novel coronavirus.

Segment Analysis

Enzyme Replacement Therapy segment is expected to hold the largest market share in the hunter syndrome treatment market.

The enzyme replacement therapy accounted for the largest market share in 2021. Hunter Syndrome is an X-linked lysosomal storage disorder due to the deficit of iduronate 2-sulfatase, an enzyme catalyzing the degradation of the glycosaminoglycans (GAG) dermatan- and heparan sulfate. The disease is mainly treated by Enzyme Replacement Therapy (ERT) with idursulfase, which has been used since 2006. Several products in ERT sales have significantly increased, which results in its growth. For instance, On January 22, 2021, GC Pharma (previously known as Green Cross Corporation) and Clinigen K.K. received manufacturing and marketing authorization for Hunterase ICV (intracerebroventricular) Injection of 15 mg (generic name: idursulfase-beta (recombinant)) as a therapy for mucopolysaccharidosis type II (Hunter syndrome). In Japan and other countries, an intravenous enzyme replacement medication is already used to treat the systemic symptoms of mucopolysaccharidosis type II. However, no therapeutic medication targets the central nervous system symptoms that affect around 70% of patients. Therefore, it has increased the demand for enzyme replacement therapy. Thus, from the above statements, the market segment accounted for the largest market share in the forecast period.

Geographical Analysis

North American region is expected to hold the largest market share in the global hunter syndrome treatment market.

North America dominates the global hunter syndrome treatment market. The higher diagnosis and treatment rates, a higher patient population and awareness of the treatment options. The presence of major clinical-stage biopharmaceutical companies as their pipeline candidates is responsible for the region's dominant share. For instance, Homology Medicines, Inc. completed a Phase 1 open-label, sequential ascending dose-escalation study in October 2022. This study aims to assess the safety and efficacy of a single IV infusion of the investigational gene therapy HMI-203. Males between the ages of 18 and 45 with MPS II (Hunter syndrome) who are currently taking idursulfase ERT (or the equivalent) are enrolled in the study. Thus, from the above statements, the North American region accounted for the largest market share in the forecast period.

Competitive Landscape

Major key players in the hunter syndrome treatment market are Denali Therapeutics Inc., Shire, AVROBIO, Green Cross Corporation, Takeda, Centogene AG Rostock, JCR Pharmaceuticals Co., Ltd., AVROBIO, ArmaGen, Inventiva and others.

Denali Therapeutics:

Overview:

Denali Therapeutics Inc. is a privately held biotechnology company focused on discovering and developing therapies for patients with neurodegenerative diseases, including Alzheimer's disease, Parkinson's disease, ALS, and others. Located in South San Francisco, investors share the vision that recent scientific insights in genetics, biology, and translational medicine offer an unprecedented opportunity to discover and develop effective medicines for neurodegenerative disease. Denali is rigorously pursuing a science-driven approach to translational medicine and clinical development.

Pipeline Product:

U.S Food and Drug Administration (FDA) have granted Fast Track designation to ETV: IDS (DNL310) for the treatment of patients with Hunter syndrome (MPS II). DNL310, Denali's lead investigational brain-penetrate enzyme replacement therapy, is under evaluation in a Phase 1/2 study in patients with Hunter syndrome as a potential treatment for both central nervous system (CNS) and peripheral manifestations of the disease. Using Denali's transport vehicle technology, a company designed DNL310 to treat both body and brain in Hunter syndrome after intravenous administration.

The global hunter syndrome treatment market report would provide an access to an approx. 45+market data table, 40+figures and 180pages.