Niemann-Pick Disease Market
Number of pages: 100 | Report Format: PDF | Published date: March 31, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023-2031
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Report Attribute |
Details |
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CAGR |
5.2% |
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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 |
Disease Type, Treatment, 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 Niemann-Pick disease market registers a revenue CAGR of 5.2% during the forecast period.
Niemann-Pick Disease Market Fundamentals
Niemann-Pick disease (NPC) is an uncommon inherited illness caused by the body's failure to move cholesterol and other lipids intracellularly, resulting in an abnormal buildup of these substances in different bodily tissues, including the brain tissue. The start age and specific symptoms of NPC can differ, from a deadly disorder within the first few months after delivery (neonatal phase) to a late-onset, chronic progressive disorder that stays undetected well into adulthood. The disease's major signs include brain and developmental disorders, hepatosplenomegaly, jaundice, cholestasis, bleeding disorders, and, in adults, swallowing and even breathing failure. Most instances are discovered in infancy and advance to life-threatening complications by the second or third decade of life. NPC is caused by changes in the NPC1 (NPC type 1C) or NPC2 (NPC type 2C) genes and is passed autosomal recessively.
There is no treatment for type A and B (NPC). The cornerstone of therapy is supportive care. Statins are used to attempt to keep blood lipid levels low, and liver processes are checked. If thrombocytopenia causes hemorrhage events, blood products may need to be transfused. Oxygen is given to ILD sufferers. Transplantation of organs has also been attempted but with limited results. Enzyme replacement treatments and gene therapies are currently being tested and may become the standard of care. Supportive care is also an essential component of treatment for type C disease. Neurological problems are treated with physical exercise. Analgesics are used to treat pain. Miglustat is a glucosylceramide synthase inhibitor that reduces glucocerebroside synthesis in Niemann-Pick and Gaucher disease. It has been authorized in Europe, Canada, and Japan but not in the United States.
NPC affects many systems and has a significant morbidity and fatality rate, whether treated or not. An interdisciplinary team comprised of a hepatologist, gastroenterologist, endocrinologist, neurologist, and genetic counselor is best suited to handle the disease. Also included in the administration are family care, nursing assistance, and social support. To handle all parts of the illness, a main expert should control patient treatment, and a nurse specialist, along with other specialists in various disciplines, should be engaged. Patient instruction is critical, as is the participation of social workers, including a geneticist. The overall incidence of lysosomal storage diseases is 1:5,000 births. Neimann-Pick-C is a neurodegenerative autosomal recessive and pan-ethnic illness with a frequency ranging from 0.35 to 2.2 per 100,000 births. The signs of Niemann-Pick-C vary based on the start age, which can be infantile, juvenile, adolescent, or adult.
The same enzymatic deficiency causes both NPA and NPB, and there is mounting proof that the two types reflect opposing ends of a spectrum. People with NPA produce little or no ASM (less than 1% of normal), whereas those with NPB produce roughly 10% of normal ASM. The activity level of an enzyme called acid sphingomyelinase (ASM) in white blood cells is used to identify NPA and NPB. The test, which requires a small blood sample from a person suspected of having the illness, is accessible at many commercial labs in the United States and elsewhere.
Niemann-Pick Disease Market Dynamics
The increasing number of collaborations regarding NPC treatment development by market participants is expected to drive market revenue growth during the forecast period. For example, Evox Therapeutics Limited, a top exosome therapeutics business, revealed a rare disease-focused collaboration with Takeda Pharmaceutical Business Limited, a multinational pharmaceutical company, in March 2020. The partnership will concentrate on developing up to five innovative protein replacement and mRNA treatments, including Evox's preclinical project in NPC type C and a second new strategy aimed at another uncommon disease that will remain unnamed.
During the forecast period, the increasing number of product clearances is expected to drive the global NPC type C therapy market revenue growth. For example, Azafaros B.V., a company developing new treatment options for patients with rare metabolic disorders such as lysosomal storage diseases (LSDs), announced in March 2022 that the U.S. Food and Drug Administration (FDA) had granted Orphan Drug Designation (ODD) for AZ-3102, a novel small molecule with a unique dual mode of action, in NPC type C. The classification was based on AZ-3120's encouraging preclinical results in an NP-C mouse model.
Clinical trial termination will likely hinder the revenue growth of the global Niemann-Pick disease type C segment during the forecast period. For example, Mandos, LLC, a biopharmaceutical firm, ended a phase II/III study in Niemann-Pick disease type C (In children, teenagers, and adults) in Australia, France, Germany, Italy, New Zealand, Singapore, Spain, Turkey, the United Kingdom, and the United States (NCT02534844) in April 2022. (EudraCT2015-002548-15). The medication was not found to be safe or useful for treating Niemann-Pick disease by the United States Food and Medication Administration (FDA).
Niemann-Pick Disease Market Ecosystem
The global Niemann-Pick disease market has been analyzed from the following perspectives: disease type, treatment, and region.
Niemann-Pick Disease Market by Disease Type
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The global Niemann-Pick disease market has been segmented based on disease type: type A, type B, type C, and type E.
Type C is expected to rise with lucrative revenue growth during the forecast period. Niemann-Pick type C is a rare neurodegenerative lysosomal storage disease that can result in a worsening mental condition characterized by supranuclear vertical gaze palsy and a movement disorder. Recent advancements in testing have made identification simpler, as have new treatments aimed at stabilizing the disease process. A novel biochemical test that measures serum cholesterol metabolites replaces skin biopsy and is more useful and reliable. Although there is no cure for this disease, miglustat, a substrate reduction therapy, has been found as a therapeutic choice in cohort studies and randomized controlled trials. The medication slows or stops disease progression in some patients. It is the first drug to demonstrate animal and clinical evidence showing a disease-modifying benefit for Niemann-Pick type C. According to consensus guidelines, the drug should be offered to all patients unless they have profound dementia requiring 24-hour care, cannot walk without a wheelchair, have a complete lack of verbal communication, or have swallowing difficulties severe enough to necessitate feeding through a percutaneous endoscopic gastrostomy.
Niemann-Pick Disease Market by Treatment
Based on disease type, the global Niemann-Pick disease market is segmented into Drug, Physical Therapy. The drug segment is further sub-segmented into Miglustat, Xenpozyme, and others.
The drug segment showed lucrative growth in terms of revenue share during the forecast period. The current therapy for NPC is miglustat, a glucosylceramide synthase (GCS) inhibitor that delays the start of neurological symptoms caused by NPC. Miglustat is the only approved medication in this area at the moment, and it can be recommended for treating neurological illnesses caused by NPC but not for treating other physiological symptoms such as hepatosplenomegaly, dystonia, and dysphagia. There are two brands of miglustat treatments available across the 3MM, with Johnson and Johnson's Zavesca and generic miglustat both available in the U.S. market. Still, neither is authorized to treat NPC, so doctors administer this therapy off-label. In Germany and the United Kingdom, two miglustat treatments are available: Piramal Enterprises' (India) Yargesa and J&J's Zavesca.
Physical therapy is expected to show the fastest revenue growth during the forecast period. Physical therapists are trained and licensed movement specialists, according to the American Physical Therapy Association (APTA). They can identify and manage a wide variety of injuries, disabilities, and illnesses. Physical therapists help enhance a person's range of motion and quality of life while preventing further harm or disability. Depending on their specialization, physical therapists can provide supplementary therapy for various medical conditions. Although physical therapists do not directly and autonomously handle medical conditions other than pure musculoskeletal conditions, they work to improve healing or teach people how to improve their movement patterns.
Niemann-Pick Disease Market by Region
Based on disease type, the global Niemann-Pick disease market is segmented into North America, Europe, Asia Pacific, Latin America, Middle East & Africa.
Because of increased research & developmental efforts, technical advancements, and greater adoption of the relevant drugs along with better diagnostic rates, North America accounts for the greatest market revenue share. Europe is also expected to expand exponentially due to an increase in cases of Niemann-Pick disease, which affects roughly 0.1 in every 10,000 individuals. The increased Niemann-Pick disease research and development is supporting further expansion. For instance, IntraBio Inc., a private pharmaceutical company, revealed favorable findings for the Phase II clinical trial, which is being conducted to evaluate the safety and efficacy of drug candidate IB1001 for treating NPC, on September 30, 2020. The clinical study demonstrated a reduction in the intensity of ataxia, a sign of NPC, and also met the objective for Clinical Impression of Change in intensity. (CI-CS). Furthermore, the European Union's orphan drug classification for miglustat promotes market revenue growth. Because of the growing frequency of inherited diseases and government concern for infant health, Asia Pacific is expected to account for rapid market revenue growth during the forecast period.
Competitive landscape of Niemann-Pick Disease Market
The prominent market players in the global Niemann-Pick disease market include:
Strategic Development in Niemann-Pick Disease Market
Niemann-Pick Disease (NPC) is an uncommon inherited illness caused by the body's failure to move cholesterol and other lipids intracellularly, resulting in an abnormal buildup of these substances in different bodily tissues, including brain tissue.
Europe is the key growth region in the global Niemann-Pick disease market in terms of revenue share.
The expected revenue CAGR of the global Niemann-Pick disease market during the forecast period is 5.2%
The major players operating in the global Niemann-Pick disease market include Johnson & Johnson Services Inc., Cyclo Therapeutics, Inc., and Kempharm, Inc.
The drug therapy segment dominates the global Niemann-Pick disease market.
*Insights on financial performance are subject to the availability of information in the public domain