Mucopolysaccharidosis Type I Market
Number of pages: 100 | Report Format: PDF | Published date: March 13, 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 |
|
CAGR |
7.5% |
|
Base Year for Estimation |
2022 |
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Forecast Period |
2023-2031 |
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Historical Year |
2021 |
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Segments Covered |
Treatment Type, End-user, and Region |
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Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
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.
Mucopolysaccharidosis Type I Market Fundamentals
Mucopolysaccharidosis type I (MPS) is a 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 various 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 MPS 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, MPS 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 transplant (HSCT) are two treatments for PMS that are frequently used in clinical settings. The disease progression may be slowed or stopped with therapy if MPSs are detected early. The primary emphasis of MPS I care before the development of 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, such as heart, pulmonology, anesthesiology, orthopedics, physiatry, otorhinolaryngology, ophthalmology, and neurosurgery. Even after creating specific treatments, this strategy that 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.
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Mucopolysaccharidosis Type I Market Dynamics
The increased prevalence of the MPS I disease is the most important driving factor for the global mucopolysaccharidosis type I market revenue growth. The male and female population is equally affected by MPS 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, such as autosomal recessive MPS I, have varying expressivity, progression, severity, and turn, 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 develop complex diseases, including MPS I, which can be carried forward over generations.
Enzyme replacement therapy is the most important factor driving the global mucopolysaccharidosis type I market revenue growth as 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. Various types of MPS serve as the leading cause for the pipeline potential in developing better treatments for an increasing number of rare illnesses. Clinical trials remain the primary emphasis of the global mucopolysaccharidosis type I market. Academic researchers, medical professionals, and top industry players are putting greater effort into creating new therapy options that can greatly alleviate symptoms and enable patients to lead normal lives as rare illnesses rise. Almost 13 drugs and biological therapeutics are in the pipeline for treating MPS I, 3, and 2 in phase II and IV trials, respectively.
However, poor diagnoses and high treatment costs are anticipated to impede market revenue growth. Also, the delay in diagnosing illnesses is another significant limiting factor for the global mucopolysaccharidosis type I market revenue growth. Additionally, finding a suitable donor is necessary for HSCT, which could significantly lengthen the process and lessen its potential advantages for which the treatment procedure gets postponed sometimes.
Mucopolysaccharidosis Type I 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
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Based on the treatment type, the global mucopolysaccharidosis type I market is segmented as enzyme replacement therapy and allogeneic HSCT. The 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 usage of enzyme replacement therapy in individuals with Gaucher disease prompted searching for a comparable treatment for other lysosomal storage diseases. MPS I was the first mucopolysaccharidosis treated with enzyme replacement therapy. It was later authorized for MPS VI, and MPS IIMPS I is treated by administering laronidase, a protein similar to human α-iduronidase that is produced via genetic engineering in a Chinese hamster ovary (CHO) cell expression system, through intravenous injection. Enzyme replacement therapy with laronidase was authorized for patient therapy in the United States by Food and Drug Administration (FDA) in 2003. 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, specialty clinics, home infusion, and others.
The hospitals segment 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 as they have all the medications required to deliver 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. Hospitals with sufficient funding can access 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 growth in 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 driving factors for the revenue growth of the North America market. Also, more patient awareness of enhanced treatment options, more sophisticated diagnostics, substantial clinical trials, and big biopharmaceutical companies with pipeline prospects are key factors attributed to the region’s larger market revenue share.
The second-largest revenue share is expected to come from the Europe market. The presence of important items in the area spurred market expansion. Asia Pacific is expected to 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 region.
Mucopolysaccharidosis Type I Market Competitive Landscape
Key companies operating in the market are expected to concentrate on securing clearances for their advanced medicines, which is expected to drive 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 market growth. Market participants are concentrating more on adopting inorganic growth strategies such as alliance and collaboration to strengthen their positions.
The prominent companies holding the largest revenue share of the global mucopolysaccharidosis type I market are:
Mucopolysaccharidosis Type I Market Strategic Developments
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 high cost of the treatment is the major restraining factor for the global mucopolysaccharidosis type I market revenue growth.
Key companies operating the global mucopolysaccharidosis type I market are BioMarin Pharmaceutical Inc., Talaris Therapeutics, Inc., JCR Pharmacspeciality Clinicseuticals Co., Ltd., Immusoft Corporation, and REGENXBIO Inc.
COVID-19 influenced the economy in three 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
Frequently Asked Questions About This Report
Key companies operating the global mucopolysaccharidosis type I market are BioMarin Pharmaceutical Inc., Talaris Therapeutics, Inc., JCR Pharmacspeciality Clinicseuticals Co., Ltd., Immusoft Corporation, and REGENXBIO Inc.
What will be the revenue CAGR of the mucopolysaccharidosis type I market during the forecast period?
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.