Hereditary Hemorrhagic Telangiectasia (HHT) - Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026

Hereditary Hemorrhagic Telangiectasia (HHT) Emerging Therapy and TPP Insights

Thelansis's "Hereditary Hemorrhagic Telangiectasia (HHT) 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.

Hereditary Hemorrhagic Telangiectasia (HHT) Overview

Hereditary hemorrhagic telangiectasia (HHT), or Osler-Weber-Rendu disease, is an autosomal dominant vascular dysplasia driven by mutations in genes like ENG or ACVRL1, altering transforming growth factor-beta (TGF-b) signaling. This causes localized disruption of vascular endothelial integrity, producing direct, fragile connections between arteries and veins without an intervening capillary bed. Patients present with recurrent epistaxis, mucocutaneous telangiectasias, and visceral arteriovenous malformations (AVMs) primarily affecting the pulmonary, hepatic, and cerebral circulations, placing them at risk for high-output heart failure, stroke, or hemorrhage. Diagnosis is established using the Curacao criteria, requiring epistaxis, telangiectasias, visceral lesions, and an affected first-degree relative. In 2026, standard management utilizes systemic antifibrinolytics like tranexamic acid, local ablative interventions, and targeted anti-angiogenic therapy with the VEGF inhibitor bevacizumab, alongside proactive screening for visceral AVMs.

Key Highlights

• In the United States, incident HHT cases are projected to increase from 4,675 in 2025 to 5,001 by 2035.
• HHT is a rare inherited vascular disorder characterized by recurrent bleeding and arteriovenous malformations.
• The disease is associated with significant clinical burden and risk of serious organ-related complications.
• Significant unmet need remains for therapies that address the underlying vascular abnormalities.

Market Overview

• The Germany HHT market is projected to grow from $22M in 2025 to $55M by 2035.
• Market growth is driven by:
• Increasing disease awareness and genetic testing
• Advancements in targeted therapeutic development
• Future growth will depend on the availability of effective disease-modifying therapies.

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
• Vaderis Therapeutics AG
• Diagonal Therapeutics, Inc.
• Atavistik Bio, Inc.