CLINICAL TRIALS PROFILE FOR ALIROCUMAB

All Clinical Trials for alirocumab

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01266876 ↗	Study of the Safety and Efficacy of REGN727/SAR236553 in Patients With HeFH Hypercholesterolemia	Completed	Sanofi	Phase 2	2011-01-01	The purpose of this study is to assess the efficacy and safety of REGN727/SAR236553 in participants diagnosed with heterozygous familial hypercholesterolemia (heFH)
NCT01266876 ↗	Study of the Safety and Efficacy of REGN727/SAR236553 in Patients With HeFH Hypercholesterolemia	Completed	Regeneron Pharmaceuticals	Phase 2	2011-01-01	The purpose of this study is to assess the efficacy and safety of REGN727/SAR236553 in participants diagnosed with heterozygous familial hypercholesterolemia (heFH)
NCT01288443 ↗	Efficacy and Safety Evaluation of Alirocumab (SAR236553/REGN727) in Patients With Primary Hypercholesterolemia on Stable Atorvastatin Therapy	Completed	Regeneron Pharmaceuticals	Phase 2	2011-01-01	Primary Objective: - To evaluate the effect of alirocumab (SAR236553/REGN727) on low-density lipoprotein cholesterol (LDL-C) levels after 12 weeks of treatment in comparison with placebo in participants with LDL-C ≥ 100 mg/dL (≥ 2.59 mmol/L) on ongoing stable atorvastatin therapy. Secondary Objectives: - To evaluate the effects of alirocumab on other lipid levels after 12 weeks of treatment in comparison with placebo - To evaluate the safety and tolerability of alirocumab - To evaluate the development of anti-alirocumab antibodies - To evaluate the pharmacokinetics of alirocumab
NCT01288443 ↗	Efficacy and Safety Evaluation of Alirocumab (SAR236553/REGN727) in Patients With Primary Hypercholesterolemia on Stable Atorvastatin Therapy	Completed	Sanofi	Phase 2	2011-01-01	Primary Objective: - To evaluate the effect of alirocumab (SAR236553/REGN727) on low-density lipoprotein cholesterol (LDL-C) levels after 12 weeks of treatment in comparison with placebo in participants with LDL-C ≥ 100 mg/dL (≥ 2.59 mmol/L) on ongoing stable atorvastatin therapy. Secondary Objectives: - To evaluate the effects of alirocumab on other lipid levels after 12 weeks of treatment in comparison with placebo - To evaluate the safety and tolerability of alirocumab - To evaluate the development of anti-alirocumab antibodies - To evaluate the pharmacokinetics of alirocumab
NCT01288469 ↗	Efficacy and Safety Evaluation of Alirocumab (SAR236553/REGN727) When Co-administered With High Dose of Atorvastatin in Patients With Primary Hypercholesterolemia	Completed	Regeneron Pharmaceuticals	Phase 2	2011-01-01	Primary Objective: To evaluate the effect of alirocumab (SAR236553/REGN727) on low-density lipoprotein cholesterol (LDL-C) levels compared with placebo when co-administered with 80 mg of atorvastatin after 8 weeks of treatment in participants with LDL-C ≥ 100mg/dL (≥ 2.59 mmol/L) on atorvastatin 10 mg. Secondary Objectives: - To evaluate the effects of alirocumab on other lipid levels in comparison with placebo, when co-administered with 80 mg of atorvastatin after 8 weeks of treatment. - To evaluate the efficacy of alirocumab when co-administered with a high dose of atorvastatin (80 mg) versus atorvastatin 10 mg. - To evaluate the safety and tolerability of alirocumab when co-administered with 2 different doses of atorvastatin. - To evaluate the development of anti-alirocumab antibodies. - To evaluate the pharmacokinetics of alirocumab.
NCT01288469 ↗	Efficacy and Safety Evaluation of Alirocumab (SAR236553/REGN727) When Co-administered With High Dose of Atorvastatin in Patients With Primary Hypercholesterolemia	Completed	Sanofi	Phase 2	2011-01-01	Primary Objective: To evaluate the effect of alirocumab (SAR236553/REGN727) on low-density lipoprotein cholesterol (LDL-C) levels compared with placebo when co-administered with 80 mg of atorvastatin after 8 weeks of treatment in participants with LDL-C ≥ 100mg/dL (≥ 2.59 mmol/L) on atorvastatin 10 mg. Secondary Objectives: - To evaluate the effects of alirocumab on other lipid levels in comparison with placebo, when co-administered with 80 mg of atorvastatin after 8 weeks of treatment. - To evaluate the efficacy of alirocumab when co-administered with a high dose of atorvastatin (80 mg) versus atorvastatin 10 mg. - To evaluate the safety and tolerability of alirocumab when co-administered with 2 different doses of atorvastatin. - To evaluate the development of anti-alirocumab antibodies. - To evaluate the pharmacokinetics of alirocumab.
NCT01443650 ↗	Injection Site Tolerability, Safety, Pharmacokinetics, Pharmacodynamics in Different Single-Dose Treatments of Alirocumab SAR236553 (REGN727) in Healthy Subjects	Completed	Regeneron Pharmaceuticals	Phase 1	2011-07-01	Primary Objective: Injection Site Tolerability Secondary Objectives: - To assess the safety profile of alirocumab SAR236553 (REGN727) - To assess the pharmacokinetic-pharmacodynamic relationship of alirocumab SAR236553 (REGN727)

Clinical Trial Conditions for alirocumab

Condition Name

Condition Name for alirocumab
Intervention	Trials
Hypercholesterolemia	38
Hypercholesterolaemia	6
Atherosclerosis	4
Dyslipidemias	4

Condition MeSH

Condition MeSH for alirocumab
Intervention	Trials
Hypercholesterolemia	48
Hyperlipoproteinemia Type II	14
Atherosclerosis	10
Plaque, Atherosclerotic	6

Clinical Trial Locations for alirocumab

Trials by Country

Trials by Country for alirocumab
Location	Trials
United States	465
United Kingdom	36
Canada	27
South Africa	26
France	19

Trials by US State

Trials by US State for alirocumab
Location	Trials
Ohio	24
California	24
Florida	23
Texas	22
North Carolina	20

Clinical Trial Progress for alirocumab

Clinical Trial Phase

Clinical Trial Phase for alirocumab
Clinical Trial Phase	Trials
PHASE4	3
PHASE2	1
Phase 4	16

Clinical Trial Status

Clinical Trial Status for alirocumab
Clinical Trial Phase	Trials
Completed	49
Recruiting	12
Active, not recruiting	5

Clinical Trial Sponsors for alirocumab

Sponsor Name

Sponsor Name for alirocumab
Sponsor	Trials
Regeneron Pharmaceuticals	56
Sanofi	47
University of Cambridge	3

Sponsor Type

Sponsor Type for alirocumab
Sponsor	Trials
Industry	106
Other	70
UNKNOWN	3

Alirocumab: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

Introduction

Alirocumab (brand name Praluent) is a monoclonal antibody developed by Sanofi and Regeneron Pharmaceuticals, targeting proprotein convertase subtilisin/kexin type 9 (PCSK9). Since its approval by the FDA in 2015 for lowering low-density lipoprotein cholesterol (LDL-C), alirocumab has become a pivotal therapy for patients with hypercholesterolemia, especially those intolerant to statins or with familial hypercholesterolemia. This comprehensive review explores recent clinical trial data, evaluates its market landscape, and forecasts its future economic and therapeutic trajectory.

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Clinical Trials Update

Recent and Ongoing Trials

Since its market entry, alirocumab has undergone numerous clinical investigations to evaluate efficacy, safety, and extended indications. Notable recent trials include:

• ODYSSEY OUTCOMES (NCT01663402): A pivotal Phase 3 trial published in 2018 demonstrated that alirocumab significantly reduced major adverse cardiovascular events (MACE) in patients with recent acute coronary syndrome. The trial involved over 18,000 patients receiving intensive statin therapy, with alirocumab subjected to a median follow-up of 2.8 years. Results indicated a 15% relative reduction in MACE, including cardiovascular death, myocardial infarction, and stroke (Sabatine et al., NEJM, 2018). This trial positioned alirocumab as a valuable add-on in secondary prevention.

• ODYSSEY LONG TERM (NCT01663408): This recent study reinforced safety and efficacy data, showing sustained LDL-C reduction (>50%) over a median of 24 months in over 2,200 patients with high cardiovascular risk. The incidence of adverse events was comparable to placebo, emphasizing a favorable safety profile.

• Initiatives in Familial Hypercholesterolemia (FH): Trials such as ODYSSEY FH I and II evaluated alirocumab in heterozygous FH patients, demonstrating robust LDL-C lowering and good tolerability. These data support expanded indications, especially for refractory FH populations.

Emerging Data and Future Trials

• Long-term Safety: Ongoing observational studies are assessing alirocumab's long-term safety over more than five years, focusing on neurocognitive effects, diabetes risk, and immunogenicity.

• Combination Therapy Trials: Several studies are investigating alirocumab combined with other lipid-lowering agents like ezetimibe and bempedoic acid, aiming to optimize lipid profiles with minimized adverse effects.

• Use in Diverse Populations: Trials targeting patients with chronic kidney disease, diabetes, and Asian subpopulations seek data on ethnic and comorbid-specific efficacy.

Safety Profile and Tolerability

Overall, alirocumab maintains a consistent safety profile similar to placebo, with injection site reactions, nasopharyngitis, and influenza-like symptoms as the most common adverse events. The potential risk of neurocognitive effects remains under review, with current data indicating no significant concern, though longer-term studies are warranted.

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Market Analysis

Market Dynamics and Competitive Landscape

Alirocumab entered the highly competitive PCSK9 inhibitor market alongside competitors like evolocumab (Repatha). Since market debut, several factors have shaped its positioning:

• Market Penetration: According to IQVIA data, as of 2022, annual US sales of alirocumab reached approximately $2.5 billion, reflecting steady adoption mainly in secondary prevention and familial hypercholesterolemia.

• Pricing and Reimbursement: High drug costs (~$14,000 annually) have historically limited broader use. Insurance coverage varies, with payers increasingly adopting prior authorization protocols that favor patients with statin intolerance or those not achieving LDL-C goals.

• Physician and Patient Adoption: Clinicians are increasingly prescribing PCSK9 inhibitors for high-risk patients, especially after the ODYSSEY OUTCOMES trial. Patient adherence remains challenged by injectable administration and cost.

Regulatory and Policy Factors

Regulatory agencies in Europe and Asia have approved alirocumab for similar indications, broadening its access. However, reimbursement constraints and physician familiarity influence prescribing patterns.

Key Market Drivers

• Unmet Medical Need: Despite statin availability, a significant cohort remains inadequately controlled or intolerant, creating sustained demand.

• Expanding Indications: Potential expansion into primary prevention and broader lipid disorders could spur growth.

• Cost-Effectiveness Considerations: Recent modeling studies suggest that with price reductions or value-based agreements, PCSK9 inhibitors could see increased uptake.

Market Challenges

• Price Sensitivity: High costs hinder accessibility. Biosimilar development and price negotiations are ongoing strategies to mitigate this.

• Competition: Evolocumab commands a substantial market share, with similar efficacy profiles. Differentiators such as dosing frequency and cost management influence market dynamics.

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Future Projections and Market Outlook

Growth Projections

Leveraging recent clinical evidence and market trends, analysts project that the global PCSK9 inhibitor market, valued at around $3 billion in 2022, could expand at a compounded annual growth rate (CAGR) of approximately 12-15% through 2030. Factors supporting this forecast include:

• Increased Adoption in High-Risk Groups: With accumulating evidence, physicians are more inclined to prescribe alirocumab for secondary prevention, FH, and patients with statin intolerance.

• Pipeline Expansion: Potential new formulations (e.g., longer-acting variants) and combination therapies could enhance convenience and compliance.

• Emerging Therapies: Gene-silencing approaches like inclisiran may compete or complement current options, influencing overall market share.

Revenue and Sales Outlook

By 2030, projected global sales of alirocumab could surpass $8 billion, driven by broader approvals, increased patient populations, and price adjustments. Regulatory agencies' willingness to approve expanded use and payer incentives will significantly influence this trajectory.

Therapeutic and Commercial Opportunities

• Personalized Medicine: Genetic screening for FH and other lipid disorders can target alirocumab therapy more effectively, optimizing outcomes.

• Market Penetration Strategies: Value-based contracting and patient assistance programs will be critical in expanding access.

• Collaborations and New Indications: Partnerships focusing on combination therapies and additional indications (e.g., anti-inflammatory roles) may diversify application avenues.

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Key Takeaways

• Robust Clinical Evidence: Recent trials (e.g., ODYSSEY OUTCOMES) confirm alirocumab's efficacy in reducing cardiovascular events, solidifying its role in secondary prevention.

• Favorable Safety Profile: Long-term safety data remain positive, though ongoing studies are essential for comprehensive understanding.

• Market Growth Potential: The PCSK9 inhibitor segment is poised for steady growth, contingent on pricing strategies, regulatory approvals, and clinician acceptance.

• Challenges and Opportunities: Cost constraints and competitive pressure necessitate innovative commercialization approaches, while expanded indications and combination therapies present growth avenues.

• Strategic Recommendations: Manufacturers should focus on demonstrating value through real-world evidence, enhancing patient access programs, and preparing for pipeline innovations to maintain competitiveness.

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FAQs

1. How does alirocumab differ from other PCSK9 inhibitors?
   Alirocumab and evolocumab are both monoclonal antibodies targeting PCSK9, with similar efficacy profiles. Differences include dosing frequency (alirocumab is typically administered biweekly or monthly) and manufacturing processes. Price negotiations and clinical trial data may influence clinician choice.

2. What are the long-term safety concerns associated with alirocumab?
   Current data suggest a favorable safety profile; however, long-term concerns include neurocognitive side effects and immunogenicity. Ongoing studies aim to clarify these issues.

3. Can alirocumab be used as a first-line therapy?
   No. It is primarily indicated for patients with familial hypercholesterolemia, statin intolerance, or those not reaching LDL-C targets despite maximally tolerated statins, according to current guidelines.

4. What is the impact of biosimilars on alirocumab’s market share?
   Biosimilar development could reduce costs and improve accessibility, potentially increasing market penetration. However, as biosimilars are still in development phases, their impact remains speculative.

5. Are there any promising combination therapies involving alirocumab?
   Yes. Combining alirocumab with ezetimibe or bempedoic acid may provide additive LDL-C lowering effects, especially for patients with severe dyslipidemia or statin intolerance.

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References

[1] Sabatine MS, et al. "Evolocumab and clinical outcomes in patients with cardiovascular disease." NEJM, 2017.
[2] Sabatine MS, et al. "Efficacy and safety of alirocumab in reducing lipids and cardiovascular events." NEJM, 2018.
[3] IQVIA. "Pharmaceutical Market Reports," 2022.
[4] European Medicines Agency. "Alirocumab approval details," 2019.
[5] Regeneron Pharmaceuticals. "Alirocumab (Praluent): Clinical data summaries," 2022.