원발성 고옥살산뇨증 : 신규 요법, 미충족 요구 및 TPP 인사이트 보고서(2026년)

Primary Hyperoxaluria Emerging Therapy and TPP Insights

Thelansis's "Primary Hyperoxaluria Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026" 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.

Primary Hyperoxaluria Overview

Primary hyperoxaluria (PH) is a rare inherited disorder of hepatic glyoxylate metabolism in which excess oxalate causes recurrent calcium oxalate stones, nephrocalcinosis, chronic kidney disease, and systemic oxalosis. PH1, PH2, and PH3 differ in severity, with PH1 carrying a high risk of kidney failure. Management includes high fluid intake, urine alkalinization, stone management, and dialysis or transplantation in advanced disease. The therapeutic landscape has shifted toward targeted RNA interference. Lumasiran, an RNAi therapy targeting glycolate oxidase, is FDA-approved for pediatric and adult patients with PH1 to lower urinary and plasma oxalate. Nedosiran, targeting hepatic lactate dehydrogenase A, is FDA-approved for PH1 in patients aged 9 years and older with relatively preserved kidney function. Long-term studies support sustained oxalate reduction, while substrate-reduction and gene-based strategies remain investigational. These therapies may preserve kidney function longer.

Key Highlights

• In Germany, diagnosed Primary Hyperoxaluria (PH) cases are projected to increase from 168 in 2025 to 171 by 2035.
• Primary Hyperoxaluria is a rare inherited metabolic disorder characterized by excessive oxalate production, leading to recurrent kidney stones and progressive renal damage.
• Delayed diagnosis can result in irreversible kidney dysfunction and systemic complications.
• Significant unmet need remains for therapies that prevent disease progression and preserve kidney function.

Market Overview

• The Spain Primary Hyperoxaluria market is projected to grow from $10M in 2025 to $22M by 2035.
• Market growth is driven by:
• Increasing adoption of RNA-targeted and disease-modifying therapies
• Improved genetic testing and earlier diagnosis
• Future growth will depend on therapies that effectively reduce oxalate production and improve long-term renal outcomes.

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

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• Alnylam Pharmaceuticals
• YolTech Therapeutics Co., Ltd.
• Arbor Biotechnologies
• Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Table of Contents

1. Key Findings and Analyst Commentary

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

2. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key emerging therapies - profiles and KOL insights

3. Product Attribute Analysis

• Key takeaways
• Scientific attributes
• Commercial attributes
• Product positioning

4. Primary Market Research

• Current treatment landscape
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Current challenges
• Unmet needs
• Emerging therapies
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Unmet needs and KOL expectations

5. Unmet Need and TPP Analysis

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

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

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