Niemann-Pick Type C (NPC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Niemann-Pick Type C (NPC) Market Outlook

Thelansis’s “Niemann-Pick Type C (NPC) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Niemann-Pick Type C treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Niemann-Pick Type C (NPC) Overview

Niemann-Pick Type C (NPC) is a group of disorders that involve abnormalities of intracellular transport of endocytosed cholesterol within cells, resulting in the accumulation of unesterified cholesterol in lysosomes and late endosomes. This condition is inherited in an autosomal recessive manner and caused by mutations in the NPC1 or NPC2 genes. The functions of these proteins still need to be fully understood. NPC can be classified into three subtypes based on the age of onset of neurological manifestations:

1. Visceral-neurodegenerative form: Early-infantile (< 2 years)
2. Neurodegenerative form: Late-infantile (2–6 years), Juvenile (6–15 years)
3. Psychiatric-neurodegenerative form: Adult (> 15 years)

Symptoms vary depending on the age of onset and may include jaundice, hepatosplenomegaly, ataxia, dysarthria, cognitive decline, seizures, dysphagia, and cataplexy. The differential diagnosis may consist of other lipidoses; idiopathic neonatal hepatitis and other causes of cholestatic icterus should be considered in neonates, and conditions with cerebellar ataxia, dystonia, cataplexy, and supranuclear gaze palsy in older children and adults. Apart from Zavesca in Europe, no approved therapies for managing symptoms manifest as Niemann–Pick disease.

• At least 95% of all diseases are due to mutations in the NPC1 gene and the remainder in the NPC2 gene. NPC is estimated to have a minimal incidence of 1/120 000 live births in the US. Birth prevalence estimates between 0.26 and 0.94 per 100,000 were proposed for France, the UK, and Germany.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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