Transthyretin Amyloid Cardiomyopathy (ATTR-CM) – Emerging Therapy, with Unmet Needs and TPP Insights Report – 2025
Transthyretin
Amyloid Cardiomyopathy (ATTR-CM) Emerging Therapy and TPP Insights
Thelansis’s “Transthyretin
Amyloid Cardiomyopathy (ATTR-CM) 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.
Transthyretin
Amyloid Cardiomyopathy (ATTR-CM) Overview
Transthyretin amyloid cardiomyopathy
(ATTR-CM) is a form of systemic amyloidosis characterized by misfolded
transthyretin (TTR) protein deposition in the myocardium. Another notable cause
of cardiac amyloidosis is the accumulation of immunoglobulin light-chain (AL)
aggregates. Although several amyloidogenic proteins may be deposited in various
organs and tissues, myocardial involvement is rare. Symptoms of ATTR-CM can
vary, and the condition is frequently misdiagnosed, with early stages
resembling symptoms of other conditions such as hypertensive heart failure or
hypertrophic cardiomyopathy. Some patients may be asymptomatic, while others
may progress to end-stage heart failure. The disease is classified based on the
sequence of the TTR gene, distinguishing between wtATTR-CM (no mutation) and
hATTR-CM (presence of a mutation). In 2019, the FDA approved tafamidis and
tafamidis meglumine for treating ATTR-CM. Both agents selectively bind to the
transthyretin tetramer, preventing the destabilization of the transthyretin
transport protein and the subsequent amyloid formation that leads to ATTR-CM.
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
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