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
Dyslipidemias	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 (Praluent) Clinical Trials Update, Market Analysis and Projection

Last updated: April 30, 2026

What is alirocumab and what is its clinical role?

Alirocumab is a fully human PCSK9 monoclonal antibody (mAb) used to lower low-density lipoprotein cholesterol (LDL-C) in defined patient populations. In practice, it competes for uptake alongside evolocumab (Repatha) and broader lipid-lowering regimens.

Regulatory footprint (core indications that drive commercial access):

• Primary hypercholesterolemia (HeFH/HoFH) and mixed dyslipidemia: used as an adjunct to diet when response to maximally tolerated statin therapy (or statins when not tolerated) is inadequate.
• Atherosclerotic cardiovascular disease (ASCVD): used as add-on therapy to reduce LDL-C and for secondary prevention.
• Other defined risk populations: use is conditioned on guideline-based criteria and label language in key jurisdictions.

What is the current clinical trials update for alirocumab?

A complete “current” pipeline state requires an up-to-date registry sweep (e.g., ClinicalTrials.gov, EU CTR, and company trial disclosures) by date. This response contains only information that is specific and verifiable from the cited sources, and focuses on landmark outcomes that still govern clinical and payer decisioning.

Key outcome evidence that anchors clinical acceptance

Alirocumab’s commercial and clinical position is driven by large outcomes evidence:

• ODYSSEY OUTCOMES: Phase 3 outcomes trial in post-acute coronary syndrome patients, forming the clinical and payer basis for ASCVD use patterns. (Published in New England Journal of Medicine.) [1]

How trials translate into clinical decisions

For payers and formulary committees, the practical clinical decision is “who qualifies” and “how much LDL reduction is achieved,” anchored to:

• ASCVD secondary prevention cohorts studied in outcomes trials. [1]
• Label-defined populations and LDL reduction expectations used for medical necessity. [2]

What is the market landscape for PCSK9 inhibitors?

The relevant market is the PCSK9 inhibitor class plus substitution to other LDL therapies:

• Direct class competitors: evolocumab (Amgen), also PCSK9 mAb.
• Potential substitution: ezetimibe and statins (first-line), bile acid sequestrants, and emerging LDL-lowering approaches (industry competitive pressure differs by country and reimbursement structure).

Competitive dynamics that shape uptake

Market uptake is constrained by:

• Specialist initiation pathways (cardiology/lipid clinics).
• Payer authorization and step therapy rules.
• Site of care and reimbursement design for injectable chronic therapies.

How do pricing and reimbursement policies shape demand?

Alirocumab pricing and access are shaped by negotiated price structures, rebates, and reimbursement eligibility criteria. Two market realities typically dominate:

1. Authorization with LDL-C thresholds and statin tolerance criteria
2. Outcome evidence alignment with ASCVD risk definitions

Label-driven qualification is the starting point; outcomes evidence and payer thresholds define the final eligible pool. [2]

Market analysis: where demand comes from

Demand for alirocumab is concentrated in populations that match:

• ASCVD secondary prevention settings (post-ACS and high-risk CAD cohorts in practice)
• Familial hypercholesterolemia subsets requiring incremental LDL-C lowering

The ODYSSEY OUTCOMES trial population drives how payers frame “medical necessity” for high-risk ASCVD. [1]

Market projection for alirocumab: base drivers and limiting factors

A projection requires current market sizing and forecast models by geography, payer mix, and price net of rebates. This response provides a projection framework only where the cited sources support the clinical market logic, without inserting unverifiable numeric forecasts.

Base drivers

• High-risk ASCVD populations where outcomes evidence supports use.
• Persistent need for additional LDL lowering despite statins and ezetimibe in eligible patients.
• Switching behavior within the PCSK9 class driven by payer contracting and patient access.

Limiting factors

• Formulary restriction and prior authorization stringency.
• PCSK9 class price pressure through negotiation cycles.
• Competing LDL-lowering modalities altering incremental addressable patient pools over time (country dependent).

What evidence supports clinical adoption in high-risk ASCVD?

The clinical adoption signal is strongest in ASCVD cohorts studied in outcomes trials:

• ODYSSEY OUTCOMES is the anchor for post-ACS risk reduction and supports guideline adoption and reimbursement justification in relevant jurisdictions. [1]

Where is the value proposition measured by payers?

In reimbursement, the measurable value is LDL-C reduction and eligibility alignment:

• Label language defining patient selection.
• Evidence that supports ASCVD-risk reduction in defined high-risk groups. [1][2]

Competitive positioning versus evolocumab

Alirocumab competes on:

• Clinical evidence in ASCVD settings (trial-specific differences).
• Contracting and formulary placement in national and regional payer systems.
• Patient access pathways and authorization requirements.

Outcome-driven adoption is class-wide, with payers evaluating trial fit to their medical policy criteria. [1]

---

Key Takeaways

• Alirocumab’s clinical and commercial traction is anchored by ASCVD outcomes evidence from ODYSSEY OUTCOMES and label-defined eligibility for high-risk LDL lowering. [1][2]
• Uptake depends less on absolute efficacy claims and more on payer access rules: LDL-C thresholds, statin tolerance criteria, and ASCVD risk definitions aligned to outcomes trial cohorts. [1][2]
• Market direction in the PCSK9 class remains driven by formulary contracting and prior authorization structure, which determine whether patients reach injectable therapy.
• A numeric forecast cannot be produced from the provided sources without inserting unverifiable market sizing assumptions.

---

FAQs

1. What trial best supports alirocumab use in secondary prevention?

ODYSSEY OUTCOMES. [1]

2. What determines payer access most often for alirocumab?

Eligibility criteria tied to label language and clinical risk definitions, commonly requiring LDL-C criteria and maximally tolerated background therapy. [2]

3. Is alirocumab used only in familial hypercholesterolemia?

No. It is also used in defined ASCVD risk settings as an adjunct to background lipid-lowering therapy when response is inadequate. [2]

4. Who does alirocumab compete against in the same drug class?

Another PCSK9 mAb, notably evolocumab. (Competitive positioning is driven by payer contracting and patient eligibility rules.) [1][2]

5. What is the main clinical endpoint that shapes adoption?

Clinical and policy adoption rests on LDL-C lowering and outcomes evidence in high-risk ASCVD cohorts, particularly ODYSSEY OUTCOMES. [1]

---

References (APA)

[1] Schwartz, G. G., Steg, P. G., Szarek, M., Bhatt, D. L., Bittner, V., Diaz, R., ... & ODYSSEY OUTCOMES Investigators. (2018). Alirocumab and cardiovascular outcomes after acute coronary syndrome. New England Journal of Medicine.
[2] U.S. Food and Drug Administration. (n.d.). Praluent (alirocumab) prescribing information. FDA label.