Transthyretin Amyloidosis - Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026

Transthyretin Amyloidosis Emerging Therapy and TPP Insights

Thelansis's "Transthyretin Amyloidosis 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.

Transthyretin Amyloidosis Overview

Transthyretin amyloidosis (ATTR amyloidosis) is a progressive, life-threatening systemic disease caused by misfolding and aggregation of transthyretin protein into amyloid fibrils depositing in multiple organs, occurring in hereditary form driven by pathogenic TTR gene mutations and wild-type form arising from age-related TTR instability, predominantly affecting the heart and peripheral nervous system. Cardiac involvement produces restrictive cardiomyopathy with preserved ejection fraction, progressive heart failure, conduction abnormalities, and arrhythmias, while peripheral and autonomic neuropathy characterises neurological involvement, particularly in hereditary ATTR with Val30Met and other pathogenic variants. Diagnosis integrates clinical assessment, technetium pyrophosphate scintigraphy providing non-invasive cardiac ATTR confirmation, serum and urine immunofixation excluding light-chain amyloidosis, and TTR genetic sequencing distinguishing hereditary from wild-type disease. Tafamidis is the established approved TTR stabiliser standard for cardiac ATTR. Acoramidis, demonstrating superior TTR stabilisation and meaningful survival benefit in the ATTRibute-CM trial, is currently under FDA Priority Review following resubmission, with a PDUFA target action date of November 2026, representing a highly anticipated imminent addition to the cardiac ATTR armamentarium. RNA-silencing therapies patisiran and vutrisiran, alongside antisense oligonucleotide eplontersen, substantially reduce circulating TTR production and are approved for hereditary ATTR polyneuropathy, with vutrisiran additionally approved for cardiac ATTR. Emerging gene editing approaches represent the most significant investigational frontier. Prognosis has improved substantially with disease-modifying therapy; multidisciplinary specialist care encompassing cardiology, neurology, and genetic counselling is integral to optimising long-term outcomes.

Key Highlights

• In Germany, ATTR disease burden remains significant, with Familial Amyloid Polyneuropathy (FAP) accounting for approximately 48.2% of cases and Familial Amyloid Cardiomyopathy (FAC) increasing to approximately 51.8% by 2035.
• ATTR is a progressive and potentially fatal disease characterized by transthyretin protein misfolding and amyloid deposition.
• Cardiac and neurological manifestations continue to contribute substantially to morbidity and mortality.
• Improved diagnostic capabilities are supporting earlier disease detection.
• Novel transthyretin stabilizers and gene-silencing therapies are transforming patient outcomes.

Market Overview

• The France ATTR market is projected to grow from approximately $480 MN to $1.6 BN by 2035.
• Market growth is supported by:
• Expanding adoption of transthyretin stabilizers and RNA-targeted therapies
• Improved diagnosis and disease awareness
• Market expansion is driven by premium rare disease therapies and longer treatment duration.
• Future growth will depend on next-generation gene-silencing and gene-editing approaches.

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
• Eidos Therapeutics, a BridgeBio company
• Intellia Therapeutics
• Lantheus Germany GmbH
• Pfizer
• Alexion Pharmaceuticals, Inc.