Global Niemann-Pick Disease Type C (NPC) Market - 2023-2030

Global Niemann-Pick Disease Type C (NPC) Market - 2023-2030

Global Niemann-Pick Disease Type C (NPC) Market reached US$ XX million in 2022 and is expected to reach US$ XX million by 2030, growing with a CAGR of XX % during the forecast period 2023-2030.

Niemann-Pick Disease Type C (NPC) is a rare, inherited lysosomal storage disorder that affects the metabolism of lipids (fats) within the body. It belongs to the broader category of diseases known as sphingolipidoses. The NPC1 or NPC2 genes, which are essential for the movement of lipids within cells, are the cause of NPC. These mutations cause aberrant lipid and cholesterol buildup in a variety of tissues, including the brain, liver, spleen, and lungs.

Though there is not a treatment for NPC at current, research is still going on, and numerous prospective treatments are being tested in clinical studies. The quality of life for those with NPC can be enhanced by supportive care and symptom management techniques such physical and occupational therapy, speech therapy, and medication to treat particular symptoms.

Market Dynamics: Drivers and Restraints

The strategies adopted by the companies

The strategies like partnership, acquisition and other strategies adopted by the companies accelerates the market growth. The rise of research and development activities focused on rare diseases, including NPC, contribute to the development of potential therapies and diagnostic methods.

For instance, on January 20 2022, to accelerate the finding of new therapeutic targets for Niemann-Pick disease type C, Centogene N.V. collaborated with Insilico Medicine, an end-to-end AI-driven drug discovery company, on research and development. Prior to pursuing validation in CENTOGENE's cellular models, both Companies will analyse the targets that have been identified. CENTOGENE will additionally retain the exclusive rights to any intellectual property generated from the research.

Additionally, on August 31, 2023, Zevra Therapeutics had acquired Acer Therapeutics, enhancing its portfolio of commercial products and rare diseases. With the addition of OLPRUVA, a U.S. commercial asset indicated for the treatment of UCDs and NPC, the combination is anticipated to increase and diversify Zevra's revenues as well as broaden its portfolio of rare diseases.

Moreover, the development of technology has made it possible for scientists to understand the underlying mechanisms of NPC better, particularly in the areas of genetics, proteomics, and imaging. Targeted therapeutics may be created as a result of this knowledge. Pharmaceutical companies are encouraged to create medicines for rare diseases by the regulatory organisations which provide orphan drug classification. These benefits, which promote the development of medicines for conditions like NPC, might take the form of tax credits, fee waivers, and market exclusivity. Thus, all of these factors boost market expansion.

Market Dynamics: Restraint

The market for niemann-pick disease type c (NPC) could encounter growth and development limitations due to a number of variables. Although there has been an increase in study and awareness, obstacles unique to uncommon diseases like NPC may postpone development. For instance, it demands an extensive amount of money to study and develop medications for rare diseases.

Pharmaceutical companies have a difficult time recovering these expenses due to the small patient population, which reduces the economic appeal of investing in medicines for uncommon diseases. Furthermore, reimbursement practises and insurance coverage for treatments for rare diseases might differ greatly between nations. Financial obstacles may prevent patients from receiving therapy if payment is restricted or insufficient.

Segment Analysis

The global niemann-pick disease type c (NPC) marketis segmented based on drug type, indication, age group, distribution channel and region.

The arimoclomol segment from the type segment accounted for approximately 41.7% of Niemann-Pick Disease Type C (NPC) share

For the treatment of niemann-pick disease type c (NPC), the investigational medication arimolol had proven promise in preclinical research and early-stage clinical trials. Arimoclomol's potential to address the underlying cellular pathways that contribute to the disease makes it important in the particular context of NPC. A rare and severe genetic condition, NPC.

Due to their low occurrence, uncommon diseases have few treatment options accessible. If Arimoclomol is found to be effective, it may offer NPC patients a focused medication, fulfilling a critical unmet medical need. The strategies adopted by the companies also helps boost the segment growth.

For instance, on June 22, 2023, the rights to two assets, arimoclomol, an oral treatment for Niemann-Pick disease type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin, had been purchased by XOMA Corporation from LadRx Corporation. Thus, the aforementioned variables boost segment expansion.

Geographical Analysis

North America accounted for approximately 38.4% of the market share in 2022

North America is expected to be the most lucrative region in the global niemann-pick disease Type C, owing to increasing number of product approvals from regulatory authorities. For instance, on August 21, 2023, U.S. Food and Drug Administration (FDA) has approved BGT-NPC, the company's investigational gene therapy for the treatment of NPC. Bloomsbury Genetic Therapies Limited is a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies.

Due to increased research and development efforts, improvements in medical technology, and increased awareness among healthcare professionals, Europe is expected to experience significant growth during the forecast period. These factors could result in more precise and timely diagnoses of Niemann-Pick Disease Type C treatment.

COVID-19 Impact Analysis

The chance of developing a serious illness from COVID-19 is increased in people with underlying medical disorders, especially those with uncommon diseases like NPC. The virus may be more difficult to eradicate in people with weakened immune systems or respiratory problems. Due to the rise in COVID-19 cases during the pandemic, healthcare systems all around the world under stress.

This strain may affect how quickly patients with uncommon diseases may receive the appropriate medical attention and treatments, which may have an impact on how well those with NPC manage their disease. Support services, such as physical therapy, occupational therapy, and social assistance, are crucial for many people with uncommon disorders. The general wellbeing of people with NPC may be affected if these services are interrupted because of COVID-19 restrictions.

Market Segmentation

By Drug Type
• Cyclodextrin
• Arimoclomol
• Miglustat (Zavesca)
• Others

By Indication
• Niemann-Pick Disease Drug Type C1
• Niemann-Pick Disease Drug Type C2

By Age Group
• Pediatric
• Adult

By Distribution Channel
• Hospital Pharmacies
• Online Pharmacies
• Retail Pharmacies

By Region
• North America
U.S.
Canada
Mexico
• Europe
Germany
UK
France
Italy
Spain
Rest of Europe
• South America
Brazil
Argentina
Rest of South America
• Asia-Pacific
China
India
Japan
Australia
Rest of Asia-Pacific
• Middle East and Africa

Competitive Landscape

The major global players in the market include Orphazyme, Vtesse, Inc, Johnson & Johnson, Intrabio, Cyclo Therapeutics, Inc, Bloomsbury Genetic Therapies, Kempharm, Inc, Azafaros B.V, XOMA Corporation, StrideBio, Sarepta Therapeutics, Inc and among others.

Key Developments
• In August 2021, Arimoclomol, a novel heat-shock protein amplifier, demonstrated positive results from a Phase II/III trial in Niemann-Pick disease type C (NPC).
• Arimoclomol was well tolerated; adverse events happened in 88.2% of those taking it compared to 75.0% of those taking a placebo. In comparison to placebo (31.3%), arimoclomol (14.7%) caused less major adverse events in individuals.
• On February 01, 2023, a Cooperative Research and Development Agreement (CRADA) was entered into by Scenic Biotech and the Niemann-Pick Type C (NPC) disease, a severe, fatal neurological disorder, at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), a division of the National Institutes of Health (NIH).

Why Purchase the Report?
• To visualize the global niemann-pick disease type C market segmentation based on drug type, indication, age group, distribution channel and region as well as understandkey commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of niemann-pick disease type C market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global Niemann-Pick Disease Type C market report would provide approximately 69 tables, 58 figures and 187 Pages.

Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies