Niemann-Pick Type C (NPC) Emerging Therapy and TPP Insights

Thelansis’s “Niemann-Pick Type C (NPC) Emerging Therapy, with Unmet Needs and TPP Insights Report – 2025″ provides a comprehensive analysis of the emerging competitive landscape, unmet needs, target product profiles (TPPs), trial designs, and KOL insights on key emerging therapies and key drug development opportunities in the indication.

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: Visceral-neurodegenerative form: Early-infantile (< 2 years), Neurodegenerative form: Late-infantile (2–6 years), Juvenile (6–15 years), and 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.

Geography coverage:

G8 (United States, EU5 [France, Germany, Italy, Spain, U.K.], Japan, and China)

Insights driven by surveys* with physician / key opinion leaders:

• Survey findings are corroborated and enriched by insights from interviews with leading KOLs

*Survey is customized based on client requirements

Deliverables format:

• PowerPoint presentation
• MS Excel

Key business questions answered:

• Detailed emerging competitive landscape
  • Pipeline analysis
  • Target patients for emerging therapies
  • Key companies
  • Key mechanism of actions
  • Launch date estimates, etc.
• Clinical trial landscape analysis
  • Target patient segments
  • Trial endpoints
  • Trial design
  • Recruitment criteria, etc.
• Unmet Needs and Opportunities
  • Performance of key current therapies
  • Top areas of unmet needs
  • Opportunity sizing for key unmet needs
• Target Product Profiles
  • Attributes and levels
  • Physician likelihood of prescribing
  • Expected patient shares
• KOL insights on key emerging therapies
  • Level of awareness
  • Expected use / line of therapy
  • Extent to fulfil key unmet needs
  • KOL quotes

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risk,etc.

2. Disease Context

• Disease definition, classification, etiology and pathophysiology, drug targets,etc.

3. Epidemiology

• Key takeaways
• Incidence / Prevalence
• Diagnosed and Drug-Treated populations
• Comorbidities
• Other relevant patient segments

4. Market Size and Forecast

• Key takeaways
• Market drivers and constraints
• Drug-class specific trends
• Country-specific trends

5. Competitive Landscape

• Current therapies
  • Key takeaways
  • Dx and Tx journey/algorithm
  • Key current therapies – profiles and KOL insights
• Emerging therapies
  • Key takeaways
  • Notable late-phase emerging therapies – profiles, launch expectations, KOL insights
  • Notable early-phase pipeline

6. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

7. Regulatory and Reimbursement Environments (by country and payer insights)

8. Appendix (e.g., bibliography, methodology)