Last updated: January 30, 2026
Executive Summary
Onpattro (patisiran), developed by Alnylam Pharmaceuticals, is a groundbreaking RNA interference (RNAi) therapeutic approved by the FDA in 2018 for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). This review provides a comprehensive analysis of ongoing clinical trials, market dynamics, competitive landscape, and projected growth trajectories to inform stakeholders’ strategic decisions.
Clinical Trials Update for Onpattro
Current Status of Patisiran
- Approval Timeline: FDA approval granted August 2018; EMA approval followed in August 2019 (EMA/CHMP).
- Indications: Initially approved for hereditary transthyretin amyloidosis with polyneuropathy.
- Ongoing Trials:
- APOLLO-B (NCT03997383): Evaluates efficacy and safety in cardiac ATTR amyloidosis patients.
- HELIOS-A (NCT03510704): Investigates long-term safety in polyneuropathy.
- APOLLO-CM (NCT04885464): Focuses on patients with cardiomyopathy.
- New Formulations: Trials for subcutaneous delivery and extended-release formulations are in development.
Notable Results from Recent Studies
| Trial Name |
Phase |
Population |
Key Outcomes |
Status |
| APOLLO-B |
III |
Cardiac ATTR amyloid |
Significant improvement in cardiac parameters (e.g., reduced NT-proBNP) |
Ongoing |
| HELIOS-A |
III |
Hereditary polyneuropathy |
Sustained neurological function and safety |
Ongoing |
| APOLLO-CM |
III |
Cardiac amyloid patients |
Awaiting topline data; pivotal for expanding indication |
Ongoing |
Clinical Trials Landscape
| Trial Phase |
Number of Trials |
Focus Areas |
Source |
| Phase I |
4 |
Safety, dosage, pharmacokinetics |
ClinicalTrials.gov |
| Phase II |
8 |
Efficacy, long-term safety |
ClinicalTrials.gov |
| Phase III |
5 |
Confirmatory efficacy, expanded indications |
ClinicalTrials.gov |
Market Analysis of Onpattro
Market Overview and Historical Growth
- Initial Market Entry: 2018, with rapid uptake driven by the unmet need in hereditary ATTR amyloidosis.
- Market Size (2022): Estimated $800 million, with a compounded annual growth rate (CAGR) of 18% (evaluated by IQVIA data).
- Key Regions:
- North America: Largest share (~60%)
- Europe: ~25%
- Rest of World: 15%
Drivers and Barriers
| Drivers |
Barriers |
| Orphan drug designation benefits |
High treatment cost (~$450K/year) |
| Increasing diagnosis rates |
Limited treatment awareness in some regions |
| Positive clinical trial outcomes for cardiac indications |
Competition from emerging RNAi and gene-editing therapies |
| Rising prevalence of hereditary ATTR amyloidosis |
Complex disease diagnosis processes |
Competitive Landscape
| Product |
Developer |
Approval Year |
Indication |
Mode of Action |
Market Share (2022) |
| Onpattro (patisiran) |
Alnylam Pharmaceuticals |
2018 |
Hereditary ATTR amyloidosis |
siRNA interference |
65% |
| Tegsedi (inotersen) |
Ionis Pharmaceuticals |
2018 |
Hereditary ATTR polyneuropathy |
ASO (antisense oligonucleotide) |
25% |
| vutrisiran (ALN-TTRsc02) |
Alnylam (ongoing) |
Phase III |
ATTR amyloidosis (cardiac) |
Next-gen siRNA |
- |
| Other pipeline candidates |
Various |
– |
Emerging treatments |
Gene editing, other RNA modalities |
- |
Pricing and Reimbursement Policies
- Pricing (U.S.): Approximately $450,000 per year.
- Reimbursement: Covered by Medicaid/Medicare under rare disease policies, with prior authorization typically required.
- Market Access Challenges: Cost-effectiveness analyses and pricing negotiations influence broader access.
Market Projection for Onpattro and Its Formulations
Forecast Underpins
- Assumption Basis:
- Continued expansion of approved indications.
- Regulatory approval of secondary formulations (subcutaneous, extended-release).
- Increased disease prevalence and better diagnostics.
- Competitive pressures from emerging therapies, including vutrisiran.
10-Year Market Projection (2023–2033)
| Year |
Estimated Market Size (USD billion) |
Growth Rate |
Comments |
| 2023 |
0.9 |
— |
Post-pandemic recovery, ongoing trials |
| 2025 |
1.2 |
20% CAGR |
Expanded indications, increased diagnosis rate |
| 2028 |
2.1 |
18% CAGR |
Market peaks as new formulations launch |
| 2033 |
3.5 |
15% CAGR |
Market maturity and pipeline competition |
Potential Market Expansion Factors
- Additional Indications: Cardiomyopathy, early-stage disease.
- Formulation Innovations: Subcutaneous delivery potentially improving patient compliance.
- Global Expansion: Regulatory approvals in Japan, China, and emerging markets.
Risks and Opportunities
| Risks |
Opportunities |
| Competition from vutrisiran and other modalities |
First-mover advantage in specific indications |
| High costs limiting access |
Economies of scale and biosimilar development |
| Regulatory hurdles in expanding indications |
Orphan drug exclusivity benefits prolong market rights |
Comparison with Competing Therapies
| Criterion |
Onpattro (Patisiran) |
Tegsedi (Inotersen) |
Vutrisiran (Next-gen) |
| Approval Year |
2018 |
2018 |
Pending (Phase III) |
| Delivery Method |
IV infusion |
Subcutaneous injection |
Subcutaneous injection |
| Efficacy (Neuropathy) |
Superior in some measures |
Comparable |
Expected to outperform |
| Safety Profile |
Lower risk of thrombocytopenia |
Higher (bleeding risks) |
Pending data |
| Administration Cost |
High (per infusion) |
Lower (once-weekly injection) |
Anticipated lower due to convenience |
Key Regulatory and Policy Trends
- Orphan Drug Designation: Facilitates market exclusivity in the US (7 years) and EMA (10 years).
- Pricing Strategies: Increasing pressure for value-based pricing.
- Global Access: Policies expanding coverage in emerging markets, though affordability remains a concern.
Key Takeaways
- Clinical Developments: Ongoing trials are broadening the therapeutic scope, with positive early data supporting further indications, especially in cardiac amyloidosis.
- Market Dynamics: The global ATTR amyloidosis market is rapidly growing, projected to reach over $3.5 billion by 2033.
- Competitive Landscape: Patisiran leads but faces increasing competition; next-generation therapies aim to improve efficacy, safety, and patient convenience.
- Pricing and Access: High treatment costs challenge market expansion, necessitating value-based negotiations and bio-similars.
- Future Outlook: Continued innovation and expanded indications will sustain growth, with regulatory milestones in emerging markets expected to further propel market size.
Frequently Asked Questions (FAQs)
1. What is the current clinical development status of Onpattro?
Onpattro is approved for hereditary transthyretin amyloidosis with polyneuropathy. Multiple Phase III trials are ongoing, evaluating efficacy for cardiac ATTR amyloidosis, with some extending the indications. New formulations such as subcutaneous delivery are under investigation.
2. How does Onpattro compare to competing therapies?
Onpattro remains the leading RNAi therapy owing to its early approval and efficacy. However, newer agents like vutrisiran—currently in late-stage trials—may offer comparable or superior efficacy with better safety profiles and ease of administration.
3. What is the projected market size for Onpattro in the next decade?
The global ATTR amyloidosis market is expected to grow from approximately $0.9 billion in 2023 to over $3.5 billion by 2033, driven by expanded indications, formulations, and improved diagnostics.
4. What are key challenges facing Onpatro in market expansion?
High treatment costs, limited access due to reimbursement complexities, emerging competition, and diagnostic delays pose primary challenges.
5. Are there recent regulatory changes impacting Onpattro?
Regulatory trends favor orphan drug designations and accelerated approvals. However, future policies focusing on pricing and cost-effectiveness may influence reimbursement and market access strategies.
References
[1] Alnylam Pharmaceuticals. Onpattro (patisiran) Prescribing Information, 2018.
[2] European Medicines Agency. EMA Approval of Onpattro, 2019.
[3] IQVIA Institute. 2022 Market Data on Rare Disease Treatments.
[4] ClinicalTrials.gov. Current and Ongoing Trials of Patisiran, 2023.
[5] EvaluatePharma. Global Rare Disease Market Projections, 2022.
This report provides a strategic understanding of Onpattro's clinical pipeline, market position, and future prospects, equipping healthcare stakeholders to make data-driven decisions.
