Global Niemann Pick Disease Market - 2022-2029

Global Niemann Pick Disease Market - 2022-2029

Market Overview

The global niemann pick disease market size was valued at US$ XX million in 2021 and is estimated to reach US$ XX million by 2029, growing at a CAGR of XX % during the forecast period (2022-2029).

Niemann-Pick disease is a rare inherited disease in which the body's ability to metabolize fat (cholesterol and lipids) within cells is impaired. These cells start to break down and eventually die. Niemann-Pick disease can affect the brain, nerves, liver, spleen, bone marrow, and, in severe cases, the lungs.

Market Dynamics

The factors influencing the global niemann pick disease market are the increasing research and development and growing prevalence of niemann pick disease.

The increasing research and development is expected to drive the market growth in the forecast period

Niemann-Pick disease is a rare inherited disease that affects the ability of the body to metabolize fat (cholesterol and lipids) within cells. These cells begin to malfunction and eventually die. The brain, nerves, liver, spleen, bone marrow, and, in severe cases, the lungs can all be affected by Niemann-Pick disease. Symptoms include progressive loss of nerve, brain, and other organ function in people who have this condition.

Niemann-Pick disease can strike anyone at any age, but it is most common in children. The disease has no known cure and can be fatal in some cases. The goal of treatment is to help people cope with their symptoms. Researchers at the National Institute of Neurological Disorders and Stroke (NINDS) have discovered two genes that contribute to Niemann-Pick disease type C when they are defective. The mechanisms by which lipids accumulate in these storage diseases cause harm to the body are being investigated by NINDS scientists. More research is being done to identify biomarkers (signs that can indicate disease risk and aid diagnosis) that can be used to assess Physical Therapy for lipid storage disorders. Scientists are experimenting with a compound to see if it can be used to detect Niemann-Pick type C disease in newborns. Moreover, on May 18th, 2020, Cyclo Therapeutics, Inc., a clinical-stage biotechnology company developing cyclodextrin-based products for the treatment of NPC1 and Alzheimer's disease, received positive feedback from the European Medicines Agency on its request for Scientific Advice (EMA).

The limitations associated with niemann pick disease are expected to hamper the market growth

Niemann-Pick disease has no known cure. People with type A or B diabetes have no effective treatment options. A drug called miglustat (Zavesca) may be an option for people with mild to moderate type C. An international study of 92 people with type C Niemann-Pick disease found that taking miglustat regularly for two years improved neurological symptoms. Physical therapy is an important part of the treatment process to help you keep mobility as long as possible. Because the disease progresses and symptoms worsen, people with Niemann-Pick disease should see their doctors regularly. Hence, due to all the above limitations associated with the treatment, market growth is expected to be hampered.

COVID-19 Impact Analysis

The NPC1 gene may be involved in the fight against COVID-19. Scientists have already demonstrated that the inactivation of NPC1 causes a reduction in cholesterol levels in the plasma membrane, which is required not only for effective ebolavirus infection but also for coronavirus cell entry and thus infectivity. The infectivity of type I feline coronavirus, which is very similar to SARS-CoV-2, was reduced after treatment with methyl cylcodextrine, a drug that depletes cell membrane cholesterol. NPC1 inhibitors or NPC disease mimetics could serve as anti-SARS-CoV-2 agents, according to experts. According to their findings, inhibiting the NPC1 gene could prevent COVID-19 from spreading via lipid-dependent mechanisms, which disrupt the microenvironment optimum for viral infectivity. Hence, the covid-19 is expected to have an impact on the market.

Segment Analysis

Miglustat (Zavesca) segment is expected to dominate the market growth

The only disease-specific drug approved for treating progressive neurological manifestations of Niemann-Pick disease type C (NP-C) in adult and pediatric patients is miglustat (Zavesca, Brazaves). This small iminosugar molecule reversibly inhibits glycosphingolipid synthesis. NP-C is a rare autosomal-recessive lipid storage disorder characterised by impaired intracellular lipid trafficking and progressive neurological symptoms that eventually lead to death. Treatment with oral miglustat stabilised key neurological manifestations of NP-C (including horizontal saccadic eye movement peak velocity, ambulation, manipulation, language, and swallowing) in pediatric and adult patients with the disease in a randomized clinical trial, long-term extension studies, and a retrospective observational cohort study. Other reports in the clinical experience setting have confirmed the therapeutic effects of miglustat in stabilising or slowing disease progression. Miglustat is a valuable agent for reducing the progression of clinically relevant neurological symptoms in pediatric and adult patients with NP-C in the absence of a cure, which is considered a significant achievement in the treatment of this disease.

Geographical Analysis

North America region is expected to hold the largest market share in the global niemann pick disease market

The growing prevalence of niemann pick disease and the presence of better healthcare facilities in this region are expected to drive the market growth.

NPC (Niemann-Pick disease type C) is an extremely rare, progressive neurodegenerative disease. Only about one person in a million in the United States is diagnosed with it or treated for it off-label. Moreover, the development of programs is expected to drive market growth. For instance, developing new genetic analysis methods, rapid biomarker tests, and clinical screening tools like the NPC suspicion index has improved NPC diagnosis over the last 20 years. In addition, NPC-specific assessment tools have been developed to aid in disease progression characterization. A pilot newborn screening program was announced in New York in 2019 in the hopes of increasing early diagnosis by identifying newborns with NPC. The primary goal of this study was to estimate the number of people in the United States who have an NPC diagnosis, an NPC diagnosis, are treated with off-label miglustat, and are likely to have NPC.

Competitive Landscape

With mergers, acquisitions, and product launches, the global niemann pick disease market is moderately competitive. Some of the key players in the market are Orphazyme A/S, Cyclo Therapeutics, Inc., Johnson & Johnson, Sanofi Genzyme, CTD Holdings, Inc., Okklo Life Sciences BV., Mallinckrodt Pharmaceuticals

Orphazyme A/S

Overview: Orphazyme is a late-stage biopharmaceutical company. Orphazyme is based in Denmark, with offices in the United States and Switzerland. The company was founded in 2009.

Product Portfolio: In the United States and the European Union, Arimoclomol has received Orphan Drug Designation (ODD) for NPC. The US Food and Drug Administration (FDA) has given Arimoclomol Fast-Track Designation (FTD), Breakthrough Therapy Designation (BTD), and Rare Pediatric Disease Designation (RPDD) for NPC.

Key Development: On June 17, 2021, The FDA sent Orphazyme a Complete Response Letter to its New Drug Application for arimoclomol for NPC treatment.

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