PCSK9 Inhibitors Market Size, Target Population, Competitive Landscape & Market Forecast - 2034
Key Highlights:
- PCSK9-targeted therapies block the PCSK9 protein in the liver, preventing the breakdown of LDL receptors on hepatocytes. This increases receptor availability to clear LDL (bad) cholesterol from the blood, lowering LDL-C levels by ~50-60%.
- Currently marketed PCSK9-targeted therapies are approved for treating hypercholesterolemia, mixed dyslipidemia, primary hyperlipidemia, Heterozygous and Homozygous Familial Hypercholesterolemia (HeFH, HoFH), Atherosclerotic Cardiovascular Disease (ASCVD), and for preventing myocardial infarction, stroke, unstable angina requiring hospitalization, and coronary revascularization.
- PCSK9-targeted therapies not only lower LDL-C but also reduce the risk of cardiovascular events in patients with prior heart attack, stroke, or peripheral artery disease. They are indicated for both primary prevention and secondary prevention. In practice, they are prescribed alongside statins to maximize cholesterol reduction, or as alternatives for patients who are statin-intolerant or unresponsive, particularly those at high or very high cardiovascular risk.
- PCSK9-targeted therapies innovation spans multiple molecule classes, including IgG1/IgG2 monoclonal antibodies, siRNA, small molecules, recombinant fusion proteins, and synthetic cyclic peptides.
- In 2024, the United States accounted for the largest market size for PCSK9 targeted therapies among the 7MM, accounting for approximately USD 1,766 million, which is further expected to increase by 2034.
- Among current marketed PCSK9-targeted therapies, REPATHA, PRALUENT, and LEQVIO, monoclonal antibodies (REPATHA, PRALUENT) remain the dominant class, delivering ~58-63% LDL-C reduction with proven cardiovascular benefits but limited real-world access. LEQVIO, a siRNA-based therapy, offers ~50-52% LDL-C reduction with twice-yearly dosing and growing adoption potential, especially following its FDA label expansion for statin-intolerant patients.
- The emerging pipeline for PCSK9-targeted therapies includes several potential drugs in the late and early stages, which include Enlicitide decanoate (MK-0616), Lerodalcibep (LIB-003), Laroprovstat (AZD0780), VERVE-102, and others, which can bring about significant change in the market during the forecast period.
- The PCSK9-targeted therapies pipeline is shifting toward more convenient and disruptive modalities. Lerodalcibep, a monthly SC small-binding protein, is on track for US approval in 2025. Oral candidates MK-0616 and Laroprovstat, expected in the late 2020s, aim to eliminate injection-related barriers. Longer-term, one-time gene-editing therapy VERVE-102 could replace chronic treatment models, while preclinical oral AQR-008 targets the LDLR EGF-A domain to enable sustained LDL-C clearance.
- In the United States in 2024, approximately 1.3 million individuals experience ACS each year, encompassing both myocardial infarction and unstable angina. Additionally, around 825,508 people suffer a stroke annually in 2024.
- In 2024, the total number of familial hypercholesterolemia treatment-eligible cases for PCSK9-targeted therapies in the 7MM was 325,880. Within this population, approximately 324,816 cases were attributed to HeFH, while around 1,065 cases were associated with the much rarer HoFH.
DelveInsight's "* PCSK9-targeted therapies - Target Population, Competitive Landscape, and Market Forecast - 2034" report delivers an in-depth understanding of the PCSK9-targeted therapies, historical and competitive landscape as well as the PCSK9-targeted therapies market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
The PCSK9-targeted therapies market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM PCSK9-targeted therapies market size from 2020 to 2034. The report also covers current PCSK9-targeted therapies, treatment practices/algorithms, and unmet medical needs to curate the best opportunities and assess the market's potential.
Geography Covered:
- The United States
- EU4 (Germany, France, Italy, and Spain) and the United Kingdom
- Japan
Study Period: 2020-2034
PCSK9-targeted therapies: Disease Understanding and Treatment Algorithm
PCSK9-targeted therapies Overview
PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) is a serine protease that regulates cholesterol levels by binding to LDL receptors on hepatocytes and promoting their degradation, which reduces the liver's ability to clear LDL-C ("bad" cholesterol) from the blood. PCSK9-targeted therapies block this process, preserving LDL receptors and enabling greater clearance of LDL-C, lowering levels by up to 50-60%. Beyond cholesterol reduction, they significantly reduce cardiovascular risk in patients with prior heart attack, stroke, or peripheral artery disease, and are used in both primary and secondary prevention. Typically given alongside statins for enhanced efficacy or as an alternative for statin-intolerant or high-risk patients, PCSK9-targeted therapies represent a key advancement in cardiovascular risk management.
PCSK9-targeted therapies: Target Population
The PCSK9-targeted therapies epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total cases of selected indications, total treatment eligible cases of selected indications, and total statin-intolerant/uncontrolled LDL cases for PCK9-targeted therapies in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.
- Within the familial subtype, HeFH is considerably more prevalent than HoFH. In the US, there were approximately 229,500 diagnosed prevalent cases of HeFH and 861 diagnosed prevalent cases of HoFH in 2024.
- In the US, ACS has 1.3 million cases in 2024, which are expected to increase by 2034. Similarly, stroke also accounts for 825,508 cases in 2024 in the 7MM.
- In EU4 and the UK, the largest pool of treatment-eligible patients is represented by those with ASCVD in the secondary prevention setting, including individuals with prior myocardial infarction, stroke, or peripheral arterial disease.
- Within this pool, approximately 2.3 million cases were attributed to patients with peripheral arterial disease - treated pool, 624,930 cases were associated with ACS, including acute myocardial infarction and unstable angina - treated pool, and 617,280 cases were linked to stroke treated pool in 2024.
- In 2024, the total number of familial hypercholesterolemia treatment-eligible cases for PCSK9-targeted therapies in Japan was estimated at 49,848. Within this population, approximately 49,607 cases were attributed to HeFH, while around 241 cases were associated with the much rarer HoFH.
- In the 7MM, approximately 336,472 cases were associated with HeFH, and 1,043 with HoFH. These subgroups collectively represent a substantial treatment pool where conventional lipid-lowering strategies remain insufficient, highlighting the clinical need and commercial opportunity for PCSK9-targeted therapies.
PCSK9 Treatment
Multiple clinical studies have demonstrated the efficacy of PCSK9-targeted therapies across diverse high-risk patient populations. In HeFH, PCSK9-targeted therapies reduce LDL-C by ~60% in up to 80% of patients. Among individuals with ASCVD, LDL-C reductions of 60% have been reported, translating into a 20% relative risk reduction in major adverse cardiovascular events. In statin-intolerant patients, PCSK9-targeted therapies provide consistent LDL-C lowering of ~50%, offering a well-tolerated alternative to standard therapy. For very high-risk populations, such as those with prior myocardial infarction, stroke, or peripheral artery disease, PCSK9-targeted therapies have been associated with significant event reduction, including ~20% fewer myocardial infarctions, ~20% fewer ischemic strokes, and improved outcomes in secondary prevention settings. Across primary prevention cohorts, early data also indicate meaningful LDL-C lowering and risk reduction in high baseline LDL-C patients who are unable to achieve targets with statins alone. Collectively, PCSK9-targeted therapies demonstrate robust and consistent benefit across multiple subgroups, positioning them as a cornerstone of next-generation lipid management.
PCSK9-targeted therapies Drug Chapters
The drug chapter segment of the PCSK9-targeted therapies report encloses a detailed analysis of PCSK9-targeted therapies, marketed drugs, and late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the PCSK9-targeted therapies clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.
Marketed Drugs
PRALUENT (alirocumab): Sanofi/Regeneron Pharmaceuticals
PRALUENT is a fully humanized monoclonal antibody used for individuals with cardiovascular diseases to reduce the risk of heart attack, stroke, and certain types of chest pain conditions. It is also indicated, along with diet and statin therapy, to reduce LDL-C in adults with primary hyperlipidemia, including HeFH and HoFH. Further, it is used for children aged 8 years and older with HeFH to reduce LDL-C levels. The drug has been approved in more than 60 countries and launched in more than 30 countries, including the US, Japan, Germany, the UK, Spain, and some other countries.
REPATHA (evolocumab): Amgen
REPATHA is a human Immunoglobulin G2 (IgG2) monoclonal antibody directed against human PCSK9. REPATHA has an approximate molecular weight of 144 kDa and is produced in genetically engineered mammalian cells. It binds to PCSK9 and inhibits circulating PCSK9 from binding to the LDL receptor, preventing PCSK9-mediated LDL receptor degradation and permitting the LDL receptor to recycle back to the liver cell surface. By this inhibition, REPATHA increases the number of LDL receptors available to clear LDL from the blood, thereby lowering LDL-C levels.
Emerging Drugs
Enlicitide Decanoate: Merck Sharp & Dohme
Enlicitide decanoate is an investigational, potentially first oral PCSK9-targeted therapy designed to lower LDL-C via the same biological mechanism as currently approved monoclonal antibody injectable PCSK9-targeted therapies, but in a daily pill form. It is a novel oral macrocyclic peptide that binds to PCSK9 and inhibits the interaction of PCSK9 with LDL receptors. The global drug discovery community has voted it as their top molecule of 2023.
In June 2025, Merck announces positive top-line results from the first two Phase III CORALreef trials evaluating enlicitide decanoate for the treatment of adults with hyperlipidemia.
Lerodalcibep (LIB-003): LIB Therapeutics
Lerodalcibep is a novel, third-generation, PCSK9-targeted therapy in development to overcome the limitations of current LDL-C lowering treatments. Lerodalicibep is being developed as a convenient, small-injection volume, once-monthly dose with long-ambient stability. Combined with sustained LDL-C reductions demonstrated in clinical trials, lerodalcibep is expected to expand treatment options for the millions of patients around the world with ASCVD, and those at very high and high risk for ASCVD. Currently, the drug is being studied in Phase III of clinical trials.
LIB received a formal filing by the FDA in February with an anticipated PDUFA (Prescription Drug User Fee Act) target action date of December 12, 2025.
PCSK9-targeted therapies Market Outlook
PCSK9-targeted therapies represent a high-growth opportunity in the cardiovascular space, driven by increasing patient demand, large target population size, and a heightened sensitivity to statins, proven clinical efficacy, and a robust clinical pipeline. Although pricing pressures and competitive dynamics remain key risks, improving affordability and expanding access are expected to support sustained adoption and create significant long-term value.
The current PCSK9 market is dominated by injectable (Amgen's REPATHA). However, this dominance is expected to be challenged by Novartis's LEQVIO, which is expected to become a blockbuster in 2025. In addition to this, in the next few years launch of oral PCSK9-targeted therapies is also expected, followed by the highly anticipated approval of Verve's gene therapy. Emerging therapies (oral and gene therapy) are going to redefine cardiovascular treatment paradigms, owing to the critical advantage in a market where patients often struggle to maintain adherence to injectable therapies. Merck (enlicitide decanoate), AstraZeneca (laroprovstat), and others are developing oral PCSK9-targeted therapies; however, Merck is leading this space, as pivotal Phase III data are available for their asset. We expect oral PCSK9 therapies to expand the total PCSK9 market by two to three times.
- The market size of PCSK9-targeted therapies in the 7MM was USD 2,413 million in 2024 and is expected to grow over the forecast period (2025-2034).
- The United States is set to remain the largest and fastest-growing PCSK9-targeted therapies market, supported by relaxed access controls and early adoption of novel agents, while EU4 and the UK growth is expected to be driven by structured reimbursement frameworks. In Japan, uptake is expected to accelerate with oral launches, aligning with a strong preference for noninjectable therapies.
- REPATHA and PRALUENT are expected to experience a sales decline during the forecast period. This downward trend reflects competition from next-generation emerging therapies, including oral PCSK9-targeted therapies and pricing pressures across key markets.
PCSK9-targeted therapies Drugs Uptake
This section focuses on the uptake rate of potential approved and emerging PCSK9-targeted therapies expected to be launched in the market during 2020-2034.
PCSK9-targeted therapies Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. It also analyzes key players involved in developing targeted therapeutics.
The presence of numerous drugs under different stages is expected to generate immense opportunities for the PCSK9-targeted therapies market growth over the forecasted period.
Pipeline Development Activities
The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for PCSK9-targeted therapies and emerging therapies.
The increasing strategic collaborations among major market players to enhance the growth of their pipeline products are anticipated to drive market expansion. For example, in July 2020, AstraZeneca and Daiichi Sankyo entered into a global collaboration to jointly develop and commercialize datopotamab deruxtecan. This collaboration allowed AstraZeneca, the marketing rights for datopotamab deruxtecan, except in Japan, and Daiichi retains the responsibility for the manufacturing and supply of datopotamab deruxtecan.
KOL Views
To keep up with current and future market trends, we take Industry Experts' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on PCSK9-targeted therapies evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along challenges related to accessibility.
DelveInsight's analysts connected with 25+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the University of Southern California and others.
Their opinion helps understand and validate current and emerging therapy treatment patterns or PCSK9-targeted therapies market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
We perform qualitative and market intelligence analysis using various approaches, such as SWOT analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.
Market Access and Reimbursement
Reimbursement for PCSK9-targeted therapies has been broad in the United States and is expanding in Europe. Manufacturers offer patient assistance programs that provide free access to PCSK9 therapies for uninsured or underinsured patients meeting specific eligibility criteria. Savings programs can reduce annual out-of-pocket costs substantially, though patients remain responsible for treatment cost-sharing and office visit expenses. These programs generally exclude claims reimbursed by Medicaid, Medicare, or other federal or state healthcare programs.
In the UK, NICE has recommended PCSK9-targeted therapies for NHS use after concluding that their cost-effectiveness falls within the acceptable range and that they meet criteria as life-extending therapies by significantly lowering LDL-C and reducing cardiovascular events compared with standard care.
The report further details country-wise reimbursement and accessibility status, cost-effectiveness assessments, patient assistance initiatives that improve affordability, and insights into coverage under government prescription drug programs.
Key Updates on PCSK9-targeted therapies
- In June 2025, Merck announced positive top-line results from the first two Phase III CORALreef trials evaluating enlicitide decanoate for the treatment of adults with hyperlipidemia.
- In May 2025, LIB Therapeutics announced key results from presentations at the 2025 European Atherosclerosis Society meeting.
- In February 2025, LIB Therapeutics announced FDA acceptance of BLA for Lerodalcibep to Lower LDL-C across a broad patient population.
- In March 2025, AstraZeneca announced AZD0780 demonstrated positive outcomes in the Phase IIb PURSUIT trial.
Scope of the Report:
- The report covers a segment of key events, an executive summary, and a descriptive overview of PCSK9-targeted therapies, explaining their mechanism and therapies (current and emerging).
- Comprehensive insight into the competitive landscape, and forecasts, the future growth potential of treatment rate, drug uptake, and drug information have been provided.
- Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current landscape.
- A detailed review of the PCSK9-targeted therapies market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
- The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis, expert insights/KOL views, and treatment preferences that help shape and drive the 7MM PCSK9-targeted therapies market.
PCSK9-targeted therapies Report Insights
- PCSK9-targeted patient pool
- Therapeutic approaches
- PCSK9-targeted therapies pipeline analysis
- PCSK9-targeted therapies market size and trends
- Existing and future market opportunity
PCSK9-targeted therapies Report Key Strengths
- Ten-year forecast
- The 7MM coverage
- Key cross competition
- Drugs uptake and key market forecast assumptions
PCSK9-targeted therapies Report Assessment
- Current treatment practices
- Unmet needs
- Pipeline product profiles
- Market attractiveness
- Qualitative analysis (SWOT)
Key Questions:
- What was the PCSK9-targeted therapies' total market size, the market size by therapies, market share (%) distribution, and what would it look like in 2034? What are the contributing factors for this growth?
- Which drug is going to be the largest contributor in 2034?
- Which drug type segment accounts for the maximum PCSK9-targeted therapies sales?
- What are the pricing variations among different geographies for approved therapies?
- How has the reimbursement landscape for PCSK9-targeted therapies evolved since the first one was approved? Do patients have any access issues that are driven by reimbursement decisions?
- What are the risks, burdens, and unmet needs of treatment with PCSK9-targeted therapies? What will be the growth opportunities across the 7MM for the patient population of PCSK9-targeted therapies?
- What are the key factors hampering the growth of the PCSK9-targeted therapies market?
- What are the indications for which recent novel therapies and technologies have been developed to overcome the limitations of existing treatments?
- What is the cost burden of approved therapies on the patient?
- Patient acceptability in terms of preferred therapy options as per real-world scenarios?
- What are the country-specific accessibility issues of expensive, recently approved therapies?
Reasons to buy:
- The report will help develop business strategies by understanding the latest trends and changing dynamics driving the PCSK9-targeted therapies market.
- Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
- Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
- Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
- Detailed analysis and ranking of indication-wise current and emerging therapies under the conjoint analysis section to provide visibility around leading indications.
- Highlights of access and reimbursement policies of approved therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
- To understand key opinion leaders' perspectives on the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
- Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
Table of Contents
1. Key Insights
2. Report Introduction
3. Executive Summary
4. Key Events
- 4.1. Upcoming Key Catalysts
- 4.2. Key Conference Insights
- 4.3. Key Transactions and Collaborations
- 4.4. News Flow
5. Epidemiology and Market Forecast Methodology
6. PCSK9-targeted Therapies Market Overview at a Glance
- 6.1. Clinical Landscape Analysis (By Phase, RoA, and Molecule Type)
- 6.2. Market Share (%) Distribution by Class of PCSK9-targeted Therapies in 2024
- 6.3. Market Share (%) Distribution by Class of PCSK9-targeted Therapies in 2034
7. Background and Overview
8. Introduction
- 8.1. Genetic Mutations and Clinical Implications
- 8.2. Mechanism of PCSK9-targeted Therapies
- 8.3. Types of PCSK9-targeted Therapies
- 8.3.1. Monoclonal Antibodies (mAbs)
- 8.3.2. Small Interfering RNA (siRNA)
- 8.4. Statins Therapy vs. PCSK9-targeted therapies
- 8.5. Use of PCSK9 therapies
- 8.5.1. Hypercholesterolemia
- 8.5.1.1. Nonfamilial (Acquired) Hypercholesterolemia
- 8.5.1.2. Familial Hypercholesterolemia
- 8.5.2. ASCVD
- 8.6. Limitations of PCSK9-targeted Therapies
- 8.7. Treatment Guidelines
9. Epidemiology and Patient Population
- 9.1. Key Findings
- 9.2. Assumptions and Rationale
- 9.3. Epidemiology Scenario in the 7MM
- 9.3.1. Total Cases of Selected Indications for PCSK9-targeted Therapies in the 7MM
- 9.3.2. Total Treatment Eligible Cases of Selected Indications for PCSK9-targeted Therapies in the 7MM
- 9.3.3. Total Statin-intolerant/Uncontrolled LDL Cases for PCSK9-targeted Therapies in the 7MM
10. Marketed Drugs
- 10.1. Key Cross Competition
- 10.2. PRALUENT (alirocumab): Sanofi/Regeneron Pharmaceuticals
- 10.2.1. Drug Description
- 10.2.2. Regulatory Milestones
- 10.2.3. Other Developmental Activities
- 10.2.4. Summary of Pivotal Trial
- 10.2.5. Analyst Views
- 10.3. REPATHA (evolocumab): Amgen
- 10.3.1. Drug Description
- 10.3.2. Regulatory Milestones
- 10.3.3. Other Developmental Activities
- 10.3.4. Summary of Pivotal Trial
- 10.3.5. Clinical Development
- 10.3.5.1. Clinical trials information
- 10.3.6. Analyst Views
- 10.4. LEQVIO (Inclisiran): Novartis/Alnylam Pharmaceuticals
- 10.4.1. Drug Description
- 10.4.2. Regulatory Milestones
- 10.4.3. Other Developmental Activities
- 10.4.4. Summary of Pivotal Trial
- 10.4.5. Clinical Development
- 10.4.5.1. Clinical trials information
- 10.4.6. Analyst Views
11. Emerging Drugs
- 11.1. Key Cross Competition
- 11.2. Enlicitide Decanoate: Merck Sharp & Dohme
- 11.2.1. Product Description
- 11.2.2. Other Development Activities
- 11.2.3. Clinical Development
- 11.2.3.1. Clinical Trial Information
- 11.2.4. Safety and Efficacy
- 11.2.5. Analyst Views
- 11.3. Lerodalcibep (LIB-003): LIB Therapeutics
- 11.3.1. Product Description
- 11.3.2. Other Development Activities
- 11.3.3. Clinical Development
- 11.3.3.1. Clinical Trial Information
- 11.3.4. Safety and Efficacy
- 11.3.5. Analyst Views
- 11.4. Laroprovstat (AZD0780): AstraZeneca
- 11.4.1. Product Description
- 11.4.2. Other Development Activities
- 11.4.3. Clinical Development
- 11.4.3.1. Clinical Trial Information
- 11.4.4. Safety and Efficacy
- 11.4.5. Analyst Views
- 11.5. VERVE-102: Eli Lilly (Verve Therapeutics)
- 11.5.1. Product Description
- 11.5.2. Other Development Activities
- 11.5.3. Clinical Development
- 11.5.3.1. Clinical Trial Information
- 11.5.4. Safety and Efficacy
- 11.5.5. Analyst Views
12. PCSK9-targeted therapies: The Seven Major Market Analysis
- 12.1. Key Findings
- 12.2. Market Outlook
- 12.3. Conjoint Analysis
- 12.4. Key Market Forecast Assumptions
- 12.5. Total Market Size in the 7MM
- 12.5.1. Total Market Size by Therapies in 7MM
- 12.6. United States Market Size
- 12.6.1. Total Market Size in the United States
- 12.6.2. Total Market Size by Therapies in the United States
- 12.7. EU4 and UK Market Size
- 12.7.1. Total Market Size in EU4 and the UK
- 12.7.2. Total Market Size by Therapies in EU4 and the UK
- 12.8. Japan
- 12.8.1. Total Market Size in Japan
- 12.8.2. Total Market Size by Therapies in Japan
13. Unmet Needs
14. SWOT Analysis
15. KOL Views
16. Market Access and Reimbursement
- 16.1. The United States
- 16.2. In EU4 and the UK
- 16.2.1. Germany
- 16.2.2. France
- 16.2.3. Italy
- 16.2.4. Spain
- 16.2.5. United Kingdom
- 16.3. Japan
17. Appendix
18. DelveInsight Capabilities
19. Disclaimer
20. About DelveInsight
