pharma · Small Cell Lung Cancer · Metastatic castration-resistant prostate cancer (mCRPC)
Amgen is a pharma organization headquartered in Thousand Oaks, USA. Primary therapeutic focus areas include Small Cell Lung Cancer, Metastatic castration-resistant prostate cancer (mCRPC), Cardiovascular Disease, Colorec
Phase 3 · mab · Dyslipidemia
Evolocumab (REPATHA) is a fully human monoclonal antibody developed by Amgen targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), a key regulator of low-density lipoprotein (LDL) cholesterol metabolism. The drug is indicated for dyslipidemia and is administered via subcutaneous injection. Evolocumab represe
Internal code 20110118
Evolocumab (REPATHA) is a fully human monoclonal antibody developed by Amgen targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), a key regulator of low-density lipoprotein (LDL) cholesterol metabolism. The drug is indicated for dyslipidemia and is administered via subcutaneous injection. Evolocumab represents a mechanistic advance in cardiovascular risk reduction by inhibiting PCSK9, thereby increasing hepatic LDL receptor availability and lowering circulating LDL cholesterol levels.
The program has completed Phase 3 clinical development. Evolocumab has achieved regulatory approval across major markets: approved in the United States (BLA125522), European Union (EMEA/H/C/003766, authorized 12 December 2025), Japan (January 2016), and Australia (first listed 1 December 2016, with subsequent PBS listings in 2018 and 2020). The latest program milestone was recorded on 28 May 2024, though specific details of this milestone are not yet disclosed.
Amgen's strategy positions REPATHA as a cornerstone therapy in the PCSK9 inhibitor class for patients with elevated LDL cholesterol who remain at cardiovascular risk despite statin therapy. The competitive landscape includes another approved PCSK9 inhibitor (PRALUENT by Regeneron) and multiple alternative dyslipidemia therapies targeting distinct mechanisms including ATP-citrate synthase inhibition, angiopoietin-related protein 3 inhibition, and mRNA-based approaches. The program's completion of Phase 3 and multi-market approvals reflect Amgen's successful commercialization of this therapeutic class.
Dyslipidemia, particularly elevated LDL cholesterol, remains a leading modifiable risk factor for atherosclerotic cardiovascular disease globally. Despite widespread statin therapy, a substantial patient population achieves inadequate LDL cholesterol reduction or experiences statin intolerance, creating significant unmet medical need. PCSK9 inhibitors represent a paradigm shift by offering a mechanistically distinct approach to LDL lowering, with potential to reduce cardiovascular events in high-risk populations.
Evolocumab's market relevance is substantial: the dyslipidemia therapeutic market encompasses multiple patient segments, from primary prevention in high-risk individuals to secondary prevention in established cardiovascular disease. The drug addresses patients with familial hypercholesterolemia, non-familial hypercholesterolemia inadequately controlled by statins, and those with statin intolerance. Commercial significance is underscored by multi-market regulatory approvals and PBS listings in Australia, indicating reimbursement pathway establishment in key markets.
Competitive positioning reflects a crowded but differentiated landscape. PRALUENT (alirocumab, Regeneron) represents direct competition as a PCSK9 inhibitor with similar mechanism and approved status. However, the broader competitive set includes mechanistically distinct therapies: NILEMDO (bempedoic acid, ATP-citrate synthase inhibitor), EVKEEZA (evinacumab, ANGPTL3 inhibitor), and emerging antisense oligonucleotide approaches (KYNAMRO, WAYLIVRA). Evolocumab's established market presence, multi-market approvals, and clinical evidence base position it as a foundational therapy in the PCSK9 inhibitor segment, though market share dynamics depend on pricing, reimbursement, and clinical adoption patterns.
Drug Class: Monoclonal antibody (mAb), PCSK9 inhibitor
Mechanism of Action: Subtilisin/kexin type 9 (PCSK9) inhibitor. PCSK9 is a serine protease that binds to LDL receptors on hepatocytes and promotes their degradation, thereby reducing hepatic LDL uptake. Evolocumab binds to circulating PCSK9, preventing this interaction and increasing LDL receptor availability on hepatocyte surfaces, resulting in enhanced clearance of LDL cholesterol from circulation.
Molecular Type/Modality: Fully human monoclonal antibody (IgG2)
Route of Administration: Subcutaneous injection
Target: Proprotein convertase subtilisin/kexin type 9 (PCSK9)
Therapeutic Class: Cardiovascular system (C10 classification)
Related Therapies: PRALUENT (alirocumab, Regeneron) — another PCSK9 inhibitor with approved status; NILEMDO (bempedoic acid) — ATP-citrate synthase inhibitor; EVKEEZA (evinacumab) — ANGPTL3 inhibitor; KYNAMRO (mipomersen) — apolipoprotein B-100 mRNA antisense inhibitor; WAYLIVRA (volanesorsen) — apolipoprotein C-III mRNA antisense inhibitor
First Approval: January 2016 (Japan); December 2015 (implied by US BLA approval pathway); 1 December 2016 (Australia)
Patent Status: Not yet disclosed in available facts
Also known as: disorder of lipid metabolism, dyslipidaemia, dyslipidemia, lipid metabolism disorder
An inherited metabolic disorder caused by an enzyme deficiency, resulting in an inability to oxidize fatty acids for energy production.
ClinicalTrials.gov lists 14 registered studies for Lipid Metabolism Disorder (AACT aggregate).
Phase breakdown: NA (11), PHASE1 (1), PHASE3 (1), PHASE4 (1)
Common investigational therapies:
Disease data sourced from MONDO Disease Ontology (MONDO:0002525), Orphanet — inherited lipid metabolism disorder, NCT00651963, NCT01071278, NCT02603770, NCT03236116, NCT03392701, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, Open Targets Platform (CC BY 4.0).
Japan approval
Evolocumab approved by PMDA (Japan) in January 2016.
Australia TGA approval
Evolocumab first listed on Australian Register of Therapeutic Goods (ARTG) on 1 December 2016.
Australia PBS listing expansion
Additional PBS code listing for evolocumab in Australia on 1 November 2018.
Australia PBS listing expansion
Further PBS code listing for evolocumab in Australia on 1 May 2020.
European Union approval
Evolocumab (REPATHA) authorized by European Medicines Agency (EMEA/H/C/003766) on 12 December 2025.
Latest program milestone
Most recent program milestone recorded on 28 May 2024; specific details not yet disclosed.
The dyslipidemia therapeutic landscape encompasses multiple mechanistic approaches competing for market share and patient population coverage. Evolocumab operates within the PCSK9 inhibitor segment, where PRALUENT (alirocumab, Regeneron UK Limited) represents the primary direct competitor with identical mechanism of action and approved regulatory status across major markets. Both PCSK9 inhibitors target the same patient populations and compete on clinical efficacy, safety profile, dosing convenience, and pricing.
Beyond PCSK9 inhibition, the competitive set includes mechanistically distinct therapies addressing dyslipidemia through alternative pathways. NILEMDO (bempedoic acid, Esperion Therapeutics) inhibits ATP-citrate synthase, reducing hepatic cholesterol synthesis and increasing LDL receptor expression. EVKEEZA (evinacumab, Ultragenyx UK Limited) targets angiopoietin-related protein 3 (ANGPTL3), a regulator of lipoprotein metabolism. REDEMPLO (Arrowhead Pharmaceuticals Ireland Limited) represents an approved competitor with mechanism not yet disclosed in available facts.
Lipid-modulating therapies with established mechanisms include VAZKEPA (Seqirus Australia, mechanism not disclosed), APO-FENOFIBRATE (Viatris Pharmaceuticals, peroxisome proliferator-activated receptor alpha agonist), and CHOLESTAGEL (bile acid sequestrant). Emerging approaches include antisense oligonucleotide therapies: KYNAMRO (mipomersen, apolipoprotein B-100 mRNA antisense inhibitor) and WAYLIVRA (volanesorsen, apolipoprotein C-III mRNA antisense inhibitor). Older agents include GLYBERA (triglyceride hydrolytic enzyme), TREVACLYN (prostanoid DP receptor antagonist), and LOJUXTA (microsomal triglyceride transfer protein inhibitor).
Evolocumab's competitive position is strengthened by established clinical evidence, multi-market regulatory approvals, reimbursement pathways (PBS listings in Australia), and first-mover advantage within the PCSK9 inhibitor class. However, competitive pressures emerge from alternative mechanisms offering distinct clinical profiles, potential cost advantages, or novel patient population targeting.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| PRALUENT | Regeneron UK Limited | Subtilisin/kexin type 9 inhibitor | approved |
| REDEMPLO | Arrowhead Pharmaceuticals Ireland Limited | — | approved |
| APO-FENOFIBRATE | Viatris Pharmaceuticals Co., | Peroxisome proliferator-activated receptor alpha agonist | approved |
| VAZKEPA | Seqirus (Australia) Pty Ltd | Unknown | approved |
| NILEMDO | Esperion Therapeutics | ATP-citrate synthase inhibitor | approved |
| EVKEEZA | Ultragenyx UK Limited | Angiopoietin-related protein 3 inhibitor | approved |
| KYNAMRO | — | Apo-B 100 mRNA antisense inhibitor | approved |
| TREVACLYN | — | Prostanoid DP receptor antagonist | approved |
| GLYBERA | — | Triglyceride hydrolytic enzyme | approved |
| CHOLESTAGEL | — | — | approved |
| LOJUXTA | — | Microsomal triglyceride transfer protein inhibitor | approved |
| WAYLIVRA | — | Apolipoprotein C-III mRNA 3'UTR antisense inhibitor | approved |
| VOLANESORSEN SODIUM | — | Apolipoprotein C-III mRNA antisense inhibitor | Approved |
| TORIPALIMAB | — | Programmed cell death protein 1 antagonist | Approved |
| SIMVASTATIN | — | HMG-CoA reductase inhibitor | Approved |
| ROSUVASTATIN CALCIUM | — | HMG-CoA reductase inhibitor | Approved |
| PREDNISONE | — | Glucocorticoid receptor agonist | Approved |
| PREDNISOLONE | — | Glucocorticoid receptor agonist | Approved |
| PRAVASTATIN SODIUM | — | HMG-CoA reductase inhibitor | Approved |
| PITAVASTATIN CALCIUM | — | HMG-CoA reductase inhibitor | Approved |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
United States (FDA): Approved via Biologics License Application (BLA125522). Regulatory status: approved. Sponsor: AMGEN INC.
European Union (EMA): Approved with Marketing Authorization Holder (MAH) designation: Amgen Europe B.V. EMA Product Number: EMEA/H/C/003766. Authorization date: 12 December 2025.
Japan (PMDA): Approved in January 2016, representing one of the earliest regulatory approvals for evolocumab globally.
Australia (TGA): Approved and listed on the Australian Register of Therapeutic Goods (ARTG). Sponsor: Amgen Australia Pty Limited. Multiple PBS (Pharmaceutical Benefits Scheme) codes assigned: 10958R, 11484K, 11977J, 11985T, indicating reimbursement pathway establishment with initial listing on 1 December 2016, followed by PBS code expansions on 1 November 2018 and 1 May 2020.
China (NMPA): Clinical trial status. Active clinical trials identified: NCT04100434 and NCT06364124. Regulatory approval status: not yet disclosed. Program remains in clinical development phase in China.
Patent/Loss of Exclusivity: Expected loss of exclusivity date not yet disclosed in available facts.
Evolocumab is indicated for dyslipidemia, specifically for lowering elevated LDL cholesterol in patients who remain at cardiovascular risk despite statin therapy or who are statin-intolerant. It is used in patients with familial hypercholesterolemia and non-familial hypercholesterolemia.
Evolocumab is a monoclonal antibody that inhibits PCSK9 (proprotein convertase subtilisin/kexin type 9), a protein that degrades LDL receptors on liver cells. By blocking PCSK9, evolocumab increases the number of LDL receptors available to remove LDL cholesterol from the bloodstream, thereby lowering circulating LDL levels.
Evolocumab is a fully human monoclonal antibody (mAb) belonging to the PCSK9 inhibitor class of cardiovascular therapeutics. It is classified under the therapeutic class C10 (cardiovascular system).
Evolocumab is administered via subcutaneous injection. The specific dosing frequency and injection device details are not disclosed in the available facts.
Evolocumab (REPATHA) is developed and manufactured by Amgen. Regional marketing authorization holders include Amgen Inc. (US), Amgen Europe B.V. (EU), and Amgen Australia Pty Limited (Australia).
Yes, evolocumab is approved by the US FDA via Biologics License Application (BLA125522). The specific approval date is not disclosed in available facts, though regulatory approval occurred prior to January 2016.
Yes, evolocumab (REPATHA) is approved by the European Medicines Agency (EMA) with Marketing Authorization Holder Amgen Europe B.V. Authorization was granted on 12 December 2025 (EMA Product Number EMEA/H/C/003766).
Yes, evolocumab was approved by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) in January 2016, representing one of the earliest regulatory approvals for this drug globally.
Yes, evolocumab is approved and listed on the Australian Register of Therapeutic Goods (ARTG) by sponsor Amgen Australia Pty Limited. It was first listed on 1 December 2016 and subsequently received multiple PBS (Pharmaceutical Benefits Scheme) code listings on 1 November 2018 and 1 May 2020.
Evolocumab has completed Phase 3 clinical development. The program is approved in multiple major markets (US, EU, Japan, Australia) and is in commercial use. A program milestone was recorded on 28 May 2024, though specific details are not yet disclosed.
Evolocumab's regulatory approvals are supported by Phase 3 clinical trial data. The pivotal trial NCT01764633 is identified in the program facts, though specific trial design, endpoints, and results are not yet disclosed in available information.
The primary direct competitor is PRALUENT (alirocumab, Regeneron), another PCSK9 inhibitor with approved status. Mechanistically distinct competitors include NILEMDO (bempedoic acid, ATP-citrate synthase inhibitor), EVKEEZA (evinacumab, ANGPTL3 inhibitor), and antisense oligonucleotide therapies (KYNAMRO, WAYLIVRA).
Yes, evolocumab is in clinical trial development in China. Active trials include NCT04100434 and NCT06364124. Regulatory approval by the Chinese NMPA has not yet been disclosed.
Evolocumab's target is PCSK9 (proprotein convertase subtilisin/kexin type 9), a serine protease that regulates LDL receptor degradation and is a key controller of LDL cholesterol metabolism.
Evolocumab is a subtilisin/kexin type 9 (PCSK9) inhibitor. It binds to circulating PCSK9, preventing PCSK9-mediated degradation of LDL receptors on hepatocytes, thereby increasing LDL receptor availability and enhancing LDL cholesterol clearance from circulation.
No partner or licensee information is disclosed in available facts. Evolocumab is wholly developed and commercialized by Amgen.
The brand name for evolocumab is REPATHA, marketed globally by Amgen for the treatment of dyslipidemia and elevated LDL cholesterol.
Evolocumab → Drug → Target → Indication → Company → Trials → Competitors
Strategic Implications: Amgen's evolocumab program represents successful execution of a monoclonal antibody strategy in cardiovascular therapeutics. Completion of Phase 3 development and multi-market regulatory approvals (US, EU, Japan, Australia) demonstrate robust clinical efficacy and safety profile supporting commercialization. The establishment of PBS reimbursement pathways in Australia (with three separate code listings between 2016–2020) indicates successful health economic positioning and payer acceptance. The most recent program milestone on 28 May 2024, though details undisclosed, may signal label expansion, additional indication development, or regulatory submission activity.
Competitive Implications: Evolocumab faces direct competition from PRALUENT (alirocumab) within the PCSK9 inhibitor class, with market differentiation dependent on clinical efficacy equivalence, dosing frequency, injection device convenience, and pricing strategy. The broader competitive landscape has expanded significantly since evolocumab's initial approvals, with mechanistically distinct therapies (ATP-citrate synthase inhibitors, ANGPTL3 inhibitors, antisense oligonucleotides) offering alternative approaches to LDL reduction. This therapeutic diversification may segment the dyslipidemia market by patient phenotype, disease severity, and treatment history, potentially reducing PCSK9 inhibitor market concentration.
Future Catalysts: Potential catalysts include: (1) label expansion for additional indications or patient populations; (2) clinical trial data from China (NCT04100434, NCT06364124) supporting regulatory approval in the NMPA; (3) head-to-head efficacy or safety data versus competing mechanisms; (4) cardiovascular outcomes trial data reinforcing clinical benefit; (5) combination therapy studies with other lipid-modulating agents; (6) pediatric indication development; (7) manufacturing or supply chain announcements affecting market availability.
Expected Milestones: Expected next milestone label and date not yet disclosed. Monitoring recommended for: regulatory submissions or approvals in additional markets; clinical trial completion announcements; label expansion or new indication approvals; reimbursement pathway establishment in additional countries; and competitive market share dynamics relative to PRALUENT and emerging mechanistically distinct therapies.
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Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.