Mucopolysaccharidoses are believed to occur in approximately one out of every 25,000 births. Nonetheless, due to the frequent lack of recognition, particularly in cases of milder forms, these conditions are often underreported or incorrectly diagnosed, making it challenging to establish their accurate prevalence within the general populace.
LAS VEGAS, Oct. 24, 2023 /PRNewswire/ — DelveInsight’s Mucopolysaccharidosis Market Insights report includes a comprehensive understanding of current treatment practices, mucopolysaccharidosis emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].
Key Takeaways from the Mucopolysaccharidosis Market Report
As per DelveInsight’s analysis, the mucopolysaccharidosis market is anticipated to grow at a significant CAGR by 2032 owing to the expected launch of emerging therapies.
Leading companies such as Immusoft of CA, Inc., REGENXBIO Inc., JCR Pharmaceuticals Co., Ltd., Denali Therapeutics Inc., Shire, Takeda, Abeona Therapeutics, Inc, Ultragenyx Pharmaceutical Inc, Allievex Corporation, and others are actively working in the mucopolysaccharidosis market.
Promising mucopolysaccharidosis therapies such as Autologous Plasmablasts (B cells), RGX-111, JR-171 (lepunafusp alfa), DNL310, Idursulfase-IT in Conjunction With Elaprase, ABO-102, AX 250, and others are currently in different stages of clinical development.
In February 2022, Denali Therapeutics announced new longer-term data from an ongoing Phase I/II clinical trial of DNL310 where longer-term data in 20 patients showed sustained normalization to healthy levels of CSF heparan sulfate and improvements in markers of lysosomal function consistent with durable CNS activity, now with up to one year of intravenous dosing with DNL310.
In February 2022, Regenxbio announced additional positive interim data from Cohorts 1-3 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Hunter Syndrome.
To delve into the mucopolysaccharidosis market landscape in more detail, access the Mucopolysaccharidosis Market Report
Mucopolysaccharidosis: Overview and Treatment
Mucopolysaccharidosis (MPS), a group of inherited metabolic disorders, involves the buildup of glycosaminoglycans (GAGs) within cells throughout the body due to a deficiency in specific enzymes required for their breakdown. This accumulation results in damage to various tissues and organs. Regrettably, there is currently no cure for mucopolysaccharidosis. Treatment strategies for this condition focus on symptom management and enhancing the quality of life for affected individuals. In certain cases, therapies such as stem cell transplantation or gene therapy may be explored as potential options for mucopolysaccharidosis management.
Key Analysis of Mucopolysaccharidosis Subtypes
DelveInsight has recently released a series of epidemiology-based market reports focusing on mucopolysaccharidosis and its subtypes including Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis III, Mucopolysaccharidosis IV, and Mucopolysaccharidosis VII. These reports include a comprehensive understanding of current treatment practices, historical and forecasted patient pool, emerging drugs, market share of individual therapies, and historical and forecasted market size from 2019 to 2032 segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].
Additionally, the reports feature exhaustive analyses of prominent companies working with their emerging candidates in different stages of clinical development. Let’s deep dive into the assessment of these mucopolysaccharidosis subtypes markets individually.
Mucopolysaccharidosis I (Hurler/Scheie Syndrome) Market
Hurler/Scheie syndrome, also known as mucopolysaccharidosis type I (MPS I), is a rare genetic disorder that falls under the broader category of lysosomal storage diseases. This condition primarily affects the body’s ability to break down long chains of sugar molecules called GAGs. Hurler/Scheie syndrome exists on a spectrum, with Hurler syndrome being the severe form and Scheie syndrome being milder. The prevalence of Hurler/Scheie syndrome is quite low, with estimates suggesting that it occurs in approximately 1 in 100,000 to 1 in 500,000 live births. This rarity makes it a challenging condition to diagnose and manage.
While there is no cure for this condition, treatment primarily focuses on managing the symptoms and improving the patient’s quality of life. Enzyme replacement therapy (ERT) is a common approach, where synthetic enzymes are infused into the patient’s bloodstream to help break down the accumulated sugars. Additionally, physical therapy, occupational therapy, and surgical interventions may be recommended to address specific symptoms such as joint stiffness, organ enlargement, or impaired mobility.
DelveInsight estimated that the MPS I market is expected to grow at a significant CAGR by 2032. The limited patient population and the high unmet medical need have spurred pharmaceutical companies to invest in research and development for potential treatments. The MPS I market has witnessed a surge in novel therapies, including enzyme replacement therapies and gene therapies, aimed at addressing the underlying causes of the disease. Additionally, advancements in diagnostic techniques and increased awareness among healthcare providers have led to earlier diagnosis and intervention, further influencing the MPS I market dynamics.
Mucopolysaccharidosis I Pipeline Therapies and Companies
Autologous Plasmablasts (B cells): Immusoft of CA, Inc.
RGX-111: REGENXBIO Inc.
JR-171 (lepunafusp alfa): JCR Pharmaceuticals Co., Ltd.
For a comprehensive view of the MPS I market, check out the Mucopolysaccharidosis I Market Assessment
Mucopolysaccharidosis II (Hunter Syndrome) Market
Mucopolysaccharidosis Type II (MPS II), commonly referred to as Hunter syndrome, is a rare X-linked disorder. It primarily affects males and has a progressive nature, although the rate of progression can vary among individuals. As per DelveInsight’s estimates, the total diagnosed prevalent population of Hunter syndrome in the seven major markets was around 1,180 cases in 2022. In the case of Hunter syndrome patients in the United States, the diagnosed prevalent cases were found to be around 520 cases in 2022, which are expected to rise during the study period 2019–2032.
The treatment landscape for Hunter syndrome has evolved over time. Currently, the only FDA-approved medication for this condition is ELAPRASE (idursulfase). This drug, which originated from the research efforts of Shire (now part of Takeda), has been a key player in managing the disease. In January 2021, there was a significant development in Japan with the approval of HUNTERASE ICV (intracerebroventricular) Injection 15 mg, a generic form of idursulfase-beta (recombinant) developed by GC Pharma. This marked a milestone as the world’s first and only drug for addressing the central nervous system symptoms of mucopolysaccharidosis type II. Furthermore, in Japan, JCR Pharmaceuticals’ IZCARGO (pabinafusp Alfa) has also gained approval as a therapeutic option for Hunter Syndrome.
As per DelveInsight analysis, the Hunter syndrome market is predicted to grow from USD 400 million in 2022 at a significant CAGR by 2032 owing to the advancements in research and development, the rise in the number of prevalent cases for Hunter syndrome, the rise in awareness of the disease, along with the expected launch of emerging therapies.
Hunter Syndrome Pipeline Therapies and Companies
JR-141: JCR Pharmaceuticals Co., Ltd.
RGX-121: REGENXBIO Inc.
DNL310: Denali Therapeutics Inc.
Idursulfase-IT in Conjunction With Elaprase: Shire/Takeda
Discover more about MPS II drugs in development @ Hunter Syndrome Clinical Trials
Mucopolysaccharidosis III (Sanfilippo Syndrome) Market
Mucopolysaccharidosis III, also known as Sanfilippo syndrome, is a rare and progressive genetic disorder that falls under the category of lysosomal storage diseases. The prevalence of Mucopolysaccharidosis III varies among its subtypes, with Type A being the most common and Type D being the rarest. In general, these disorders are considered extremely rare, with an estimated combined incidence of 1 in 70,000 to 1 in 500,000 live births. It primarily affects children and leads to a wide range of symptoms, including developmental delays, neurological impairment, behavioral issues, and physical abnormalities.
The MPS III treatment approach typically involves a combination of supportive therapies, including physical therapy, occupational therapy, and speech therapy, to address developmental delays and improve mobility and communication skills. Additionally, enzyme replacement therapy (ERT) and gene therapy approaches are being explored as potential treatments to address the underlying enzyme deficiency responsible for the condition. These emerging therapies aim to slow down the progression of the disease and alleviate some of the associated symptoms. However, the management of MPS III often requires a multidisciplinary team of healthcare professionals to tailor a comprehensive care plan to meet the unique needs of each patient.
As per Delveinsight analysis, the MPS III market is expected to grow at a significant CAGR by 2032. In recent years, MPS III has garnered increased attention from pharmaceutical companies and research institutions. This heightened interest can be attributed to a growing understanding of the disease’s underlying mechanisms, improved diagnostic methods, and advancements in potential treatment options. The MPS III market is becoming more competitive as multiple stakeholders aim to develop innovative therapies that can alleviate the debilitating symptoms and improve the quality of life for affected individuals. Additionally, initiatives for rare disease awareness and advocacy have contributed to greater patient and caregiver involvement in decision-making, shaping the direction of research and development efforts.
Mucopolysaccharidosis III Pipeline Therapies and Companies
ABO-102: Abeona Therapeutics, Inc/Ultragenyx Pharmaceutical Inc
AX 250: Allievex Corporation
To gain a deeper understanding of the Sanfilippo syndrome market, be sure to explore the Mucopolysaccharidosis III Market Outlook
Mucopolysaccharidosis IV (Morquio Syndrome) Market
Mucopolysaccharidosis IV, also known as MPS IV or Morquio syndrome, is a rare and inherited metabolic disorder that falls within the broader category of lysosomal storage diseases. MPS IV is subdivided into two types, MPS IV A and MPS IV B, each caused by a deficiency in a different enzyme. This disorder is exceptionally rare, with an estimated prevalence of approximately 1 in 200,000 to 300,000 births.
Treatment for mucopolysaccharidosis IV typically involves a multidisciplinary approach aimed at managing the various symptoms and improving the patient’s quality of life. Enzyme replacement therapy has shown promise as a primary treatment option for some forms of MPS IV. ERT involves the intravenous administration of a missing or deficient enzyme, such as GALNS (N-acetylgalactosamine-6-sulfatase), which can help slow down the progression of the disease and alleviate certain symptoms. Additionally, supportive care is essential and may include orthopedic interventions, such as surgery to address skeletal deformities, physical therapy, and pain management.
As per Delveinsight analysis, the MPS IV market is expected to grow at a significant CAGR by 2032. There has been a growing recognition of the unmet medical needs of MPS IV patients, leading to increased research and development efforts in the field. The MPS IV market has witnessed a surge in the development of innovative therapies, including enzyme replacement therapies and gene therapies, aimed at addressing the underlying causes of the disease rather than just managing its symptoms. Additionally, regulatory agencies and advocacy groups have played a crucial role in accelerating drug approvals and improving patient access to these treatments. As a result, the MPS IV market is experiencing a transformation, with a heightened focus on improving patients’ quality of life and prognosis, offering new hope to those affected by this rare disorder.
Explore in-depth for a comprehensive understanding of the Morquio Syndrome Clinical Trials
Mucopolysaccharidosis VII (Sly Syndrome) Market
Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome is an extremely rare condition, with an estimated prevalence of approximately 1 in 1 million live births. Due to its rarity, it often goes undiagnosed or misdiagnosed, making awareness and early detection crucial for affected individuals. Due to its rarity, it often goes undiagnosed or misdiagnosed, making awareness and early detection crucial for affected individuals. Research and advancements in genetic testing and therapies continue to offer hope for improved management and treatment options for those living with this challenging disorder.
While there is currently no cure for MPS VII, several therapeutic approaches aim to alleviate its symptoms. ERT has shown promise in some cases, wherein a synthetic form of the missing enzyme is infused into the patient’s bloodstream to help break down accumulated substances. Additionally, supportive care, physical therapy, and surgery may be necessary to manage skeletal deformities, respiratory issues, and other complications associated with the condition. Ongoing research into gene therapy and other innovative treatments offers hope for improved outcomes and a potential cure in the future.
As per Delveinsight analysis, the MPS IV market is expected to grow at a significant CAGR by 2032. Advances in genetic diagnostics and increased awareness have led to earlier diagnosis and treatment initiation, driving Sly syndrome market growth. Moreover, the emergence of novel therapies, including enzyme replacement and gene therapies, has transformed the treatment landscape for mucopolysaccharidosis VII. These breakthroughs have spurred competition in the market and prompted investment in research and development efforts to further improve therapeutic options.
To access a complete analysis of the MPS VII market, visit Sly Syndrome Market Assessment
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SOURCE DelveInsight Business Research, LLP