Mucopolysaccharidosis Type I Market
Number of pages: 100 | Report Format: PDF | Published date: 13 March, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
|
Report Attribute |
Details |
|
Market size value in 2022 |
US$ 625 million |
|
Revenue forecast in 2031 |
US$ 1,288 million |
|
Growth Rate |
CAGR of 7.5% from 2023 to 2031 |
|
Base year for estimation |
2022 |
|
Forecast Period |
2023-2031 |
|
Historical Year |
2021 |
|
Segments covered |
Treatment Type, End-user, and Region |
|
Regional scope |
North America, Europe, Asia Pacific, and the Rest of the World (ROW) |
According to the deep-dive market assessment study by Growth Plus Reports, the global mucopolysaccharidosis type I market was valued at US$ 625 million in 2022 and is expected to register a revenue CAGR of 7.5% to reach US$ 1,288 million by 2031.
Market Fundamentals
Mucopolysaccharidosis type I (MPS) is rare genetic disease induced by a lysosomal enzyme deficiency in the glycosaminoglycan (GAG) breakdown pathway. This metabolic obstruction results in GAG accumulation in different organs and tissues of affected individuals, resulting in a multisystemic clinical picture, which may include cognitive impairment. Patients develop a variety of health issues that impact their quality of life and longevity if they are not addressed early. It has been observed that different geographic areas and cultural backgrounds have different incidence rates for mucopolysaccharidosis and each subtype. Each form of mucopolysaccharidosis is brought on by a diverse spread of mutations, primarily missense mutations. With a frequency of 1 in 100,000 live births for the Hurler phenotype and up to 1 in 800,000 live births for the Scheie phenotype, mucopolysaccharidosis type I is carried autosomal recessively like most lysosomal disorders. A distinctive facial appearance, corneal clouding, macroglossia, hearing loss, hydrocephaly, cardiopathy, breathing issues, hepatosplenomegaly, inguinal and umbilical hernia, dysostosis multiplex, restricted joint movement, and cognitive decline are among the most characteristic MPS I symptoms. Additionally, the buildup of GAGs in paraspinal tendons and rigid structures raises the risk of morbidity, posing significant dangers to the cervical spine. Patients with MPS I frequently require surgical procedures with a high risk of complications due to the participation of multiple systems and tissues. Enzyme replacement therapy and hematopoietic stem cell transplantation are two treatments for mucopolysaccharidosis, that are frequently used in clinical settings. The disease progression may be slowed or stopped with therapy if mucopolysaccharidoses are detected early enough. The primary emphasis of mucopolysaccharidosis type I care before the development of hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy was the control and prevention of complications. The interdisciplinary team providing this symptomatic and palliative treatment included various medical disciplines like heart, pulmonology, anesthesiology, orthopedics, physiatry, otorhinolaryngology, ophthalmology, neurosurgery, etc. was crucial. Even after creating particular treatments, this strategy which aims to cure patients and promote health, has been crucial. To keep these people healthy, avoid complications, and, to some extent, slow the development of the illness, speech therapists, occupational therapists, psychologists, and physical therapists are also crucial.
[57547]
Market Dynamics
The increased prevalence of the mucopolysaccharidosis type I disease is the most important driving factor of the global mucopolysaccharidosis type I market. Males and females are equally affected by mucopolysaccharidosis type I, which has a prevalence of 1 in 100,000 live births for the severe type and 1 in 500,000 for the attenuated type. Some rare genetic disorders, for example, autosomal recessive mucopolysaccharidosis type I, have varying expressivity, progression, severity, and turn that are influenced by environmental variables and gene-environment interactions. Environmental variables may alter an illness's intensity, timing, and appearance through direct or secondary processes, such as epigenomic influences, protein misfolding, enzymatic alteration, transporter activity, and mitochondrial effects. Therefore, several external factors such as irregular lifestyle, rising tobacco & alcohol consumption, and chemical exposure can lead to the development of complex diseases like mucopolysaccharidosis type I, which can be carried forward over generations.
Enzyme replacement therapy is the most important factor driving the global mucopolysaccharidosis type I market growth because there is a lack of a variety of therapeutics available to patients, resulting in the monopoly of a single form of therapy. The growing demand for advanced therapeutics due to unfulfilled requirements and improved therapy results is another important driving force. Numerous kinds of mucopolysaccharidosis are the primary driver of the pipeline prospects for the creation of improved therapies for a growing number of rare diseases. Clinical trials remain the primary emphasis of the global mucopolysaccharidosis type I market. Academic researchers, medical professionals, and top industry actors are putting greater effort into creating new therapy options that can greatly alleviate symptoms and enable patients to lead normal lives as the prevalence of rare illnesses rises. Almost thirteen drugs and biological therapeutics are present in the pipeline for treating mucopolysaccharidosis type I, three, and two in phase II and IV trials, respectively.
However, poor diagnoses and high treatment costs are anticipated to impede the market growth. Also, the delay in diagnosing illnesses is another significant limiting element for the global mucopolysaccharidosis type I market growth. Additionally, finding a suitable donor is necessary for hematopoietic stem cell transplantation, which could significantly lengthen the process and lessen its potential advantages for which the treatment procedure gets postponed sometimes.
Market Ecosystem
The global mucopolysaccharidosis type I market is analyzed from three perspectives: Treatment Type, End-user, and Region.
Mucopolysaccharidosis Type I Market by Treatment Type
[867556]
Based on the treatment type, the global mucopolysaccharidosis type I market is segmented as enzyme replacement therapy and allogeneic HSCT. Allogeneic HSCT segment is further sub-segmented as bone marrow and umbilical cord blood transplantation.
The enzyme replacement therapy segment accounted for the largest revenue share of the global mucopolysaccharidosis type I market in 2022. Enzyme replacement therapy is a procedure that involves the regular intravenous delivery of the enzyme, which is deficient in the patient. The first successful use of enzyme replacement therapy in individuals with Gaucher disease prompted searching for a comparable treatment for other lysosomal storage diseases. Mucopolysaccharidosis type I was the first mucopolysaccharidosis treated with enzyme replacement therapy. It was later authorized for MPS VI and MPS II. For mucopolysaccharidosis type I, the laronidase, a protein analogous to human α-iduronidase produced by genetic engineering in a Chinese hamster ovary (CHO) cell expression system is administered intravenously. Enzyme replacement therapy with laronidase was authorized for patient therapy in the United States by Food and Drug Administration in 2003, and in Europe it was approved by European Medicines Agency (EMEA) in 2003.
Mucopolysaccharidosis Type I Market by End-user
Based on the end-user, the global mucopolysaccharidosis type I market is segmented into hospitals, speciality clinics, home infusion, and others.
Hospitals accounted for the largest revenue share of the global mucopolysaccharidosis type I market in 2022. The market has taken the lead as hospitals obtain more funding to invest in state-of-the-art, cutting-edge care. Hospitals are the best location to receive treatment because they have all the medications required to give patients comprehensive care. The expanding accessibility of healthcare reimbursements in many developed countries and ongoing technological breakthroughs in regenerative therapeutics are the main driving forces behind the segment's growth. The availability of doctors, surgeons, and other specialists teams on staff makes the therapy process easier. Additionally, hospitals with sufficient funding have access to modern, recently developed therapy options and reasonably priced diagnostics. As a result, patients can rely on hospitals for care regardless of their financial situation.
Home infusion may be an option for patients who, after three to six months of infusion in the hospital, do not experience significant concoction reactions. The site of infusion as well as the storage and preparation of the drug must be approved by the reference center medical staff, and a professional nurse trained in this specific procedure continuously monitors the infusion and notify the referring physician regularly. Therefore, hospital bills can rapidly mount up. Infusion therapy administered at home is more affordable than care in a clinic or hospital. The patient can spend less money and less time in the hospital if they can get infusion treatment from home. Thus, the home infusion segment is also expected to account for the fastest-growing segment of the global mucopolysaccharidosis type I market.
Mucopolysaccharidosis Type I Market by Region
Based on the region, the global mucopolysaccharidosis type I market is segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa.
North America accounted for the largest revenue share of the global mucopolysaccharidosis type I market in 2022. Higher diagnosis and treatment rates for several rare diseases, along with suitable reimbursement guidelines for these treatments, are some driving factors for the growth of North America segment. Also, more patient awareness of enhanced treatment options, more sophisticated diagnostics, substantial clinical trials, and big biopharmaceutical companies with pipeline prospects are to blame for the region's larger market share.
The second-largest revenue share is expected to come from the European market. The presence of important items in the area spurred the market's expansion. Asia Pacific will account for a sizeable portion of global revenue. The mucopolysaccharidosis type I market is expected to be driven by several anticipated treatment launches and a sizable prospective patient population in the area.
Competitive Landscape
Key companies operating in the market are expected to concentrate on securing clearances for their advanced medicines, which is expected to drive the market development. Increasing product approval by regulatory authorities for improved treatment results is anticipated to push this trend. Major players' adoption of inorganic growth strategies is anticipated to fuel the market growth. Market participants are concentrating more on adopting inorganic growth strategies like alliance and collaboration to strengthen their positions. Some of the prominent companies holding the largest revenue share of the global mucopolysaccharidosis type I market are,
Strategic Development
Mucopolysaccharidosis type I (Hurler syndrome) is a rare, inherited disorder. MPS I is also known as Hurler syndrome. Children with Hurler syndrome have an abnormal accumulation of complex sugars in their cells, affecting many of their bodies' systems.
The excessive cost of the treatment is the major restraining factor for the global mucopolysaccharidosis type I market.
Key companies operating the global mucopolysaccharidosis type I market are BioMarin Pharmaceutical Inc., Talaris Therapeutics, Inc., JCR Pharmacspeciality Clinicseuticals Co., Ltd., Immusoft Corporation, REGENXBIO Inc., Orchard Therapeutics plc, Paradigm Biopharmaceuticals Ltd., and ArmaGen, Inc.
COVID-19 influenced the economy through three key ways: direct impacts on products and demand, distribution channel disruption, and financial impacts on businesses and the financial market.
The global mucopolysaccharidosis type I market is expected to grow at a revenue CAGR of 7.5% during the forecast period from 2023 to 2031.
*Insights on financial performance are subject to the availability of information in the public domain
Using the fundamental research and knowledge services capabilities of GRG Health, Growth Plus Reports may now be tailored based on client needs.
GRG Health's unique GrowthMIX strategy and comprehensive research methodology enable us to provide unique and valuable insights to our clients while uncovering trends that would not be discovered using traditional techniques.
Snehal Deshmukh
Principle Consultant
Speak to analyst