Biological Mechanism of Action: Modes, MoA & Classification
Biological Mechanisms of Action of Blockbuster Drugs
Behind every blockbuster drug like Ozempic, Humira, Keytruda, and Eliquis is a specific molecular mechanism. This page breaks down how today's top-prescribed therapies work, from the receptors they bind to the pathways they disrupt, grounded in 138 million peer-reviewed papers from Elicit's citation database.
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A drug's mechanism of action describes how it produces its therapeutic effect at the molecular, cellular, or systemic level. Understanding MOA is essential for drug development, competitive intelligence, and clinical research.
| MOA Name | Generic Drug Name | Target | Mechanism | Example Drugs | Indication |
|---|---|---|---|---|---|
| Androgen receptor inhibition | Enzalutamide | Androgen receptor (AR) | Androgen receptor (AR) signaling inhibitor / androgen receptor inhibitor | - Xtandi |
- Castration-resistant prostate cancer (CRPC) - Metastatic castration-sensitive prostate cancer - Non-metastatic CSPC with biochemical recurrence (high metastasis risk) |
| CD20-directed B-cell depletion | Ocrelizumab | CD20 (MS4A1) | CD20-directed cytolytic antibody; engagement of CD20 causes cell lysis resulting in B-cell depletion | - Ocrevus | - Relapsing MS (CIS, relapsing-remitting, active secondary progressive) - Primary progressive multiple sclerosis |
| CDK4/6 inhibition | Palbociclib | CDK4/CDK6 | Oral reversible CDK4/6 inhibitor that blocks progression of cells from G1 into S phase of the cell cycle | - Ibrance | - HR+/HER2- advanced or metastatic breast cancer (first-line + aromatase inhibitor) - HR+/HER2- advanced or metastatic breast cancer (+ fulvestrant after endocrine therapy |
| CDK4/6 inhibition | Abemaciclib | CDK4/CDK6 | Selective inhibitor of CDK4 and CDK6, inhibiting progression from the G1 into S phase of the cell cycle | - Verzenio | - HR+/HER2- early breast cancer (node-positive, high recurrence risk, Ki-67 ≥20%) - HR+/HER2- advanced or metastatic breast cancer |
| Dual GIP/GLP-1 receptor agonism | Tirzepatide | GIPR/GLP1R | Dual GIP and GLP-1 receptor agonist; increases insulin secretion and reduces glucagon levels in a glucose-dependent manner | - Mounjaro - Zepbound |
- Type 2 diabetes mellitus - Chronic weight management (obesity or overweight with comorbidities) - Obstructive sleep apnea in adults with obesity |
| Factor VIII cofactor activity mimicry | Emicizumab | FIXa (F9) and FX (F10) | Humanized bispecific antibody mimicking activated factor VIII cofactor activity by bridging activated factor IX (FIXa) and factor X (FX), restoring hemostasis | - Hemlibra | - Bleeding episode prophylaxis in hemophilia A (±factor VIII inhibitors) |
| Factor Xa inhibition | Apixaban | F10 (factor X / factor Xa) | Direct, reversible, highly selective inhibitor of factor Xa (FXa); inhibits free and clot-bound FXa to decrease thrombin generation and thrombus development | - Eliquis | - Nonvalvular atrial fibrillation (stroke/SE risk reduction) - DVT prophylaxis after hip or knee replacement - Deep vein thrombosis (DVT) treatment - Pulmonary embolism (PE) treatment - Recurrent DVT/PE risk reduction |
| Factor Xa inhibition | Rivaroxaban | F10 (factor X / factor Xa) | Factor Xa inhibitor (direct oral anticoagulant) | - Xarelto | - Nonvalvular atrial fibrillation (stroke/SE risk reduction) - Deep vein thrombosis (DVT) treatment - Pulmonary embolism (PE) treatment - Recurrent DVT/PE risk reduction - DVT prophylaxis after hip or knee replacement - VTE prophylaxis in acutely ill medical patients - Major CV event risk reduction in CAD - Major thrombotic event risk reduction in PAD - Pediatric VTE treatment and recurrence risk reduction - Pediatric thromboprophylaxis after Fontan procedure |
| GLP-1 receptor agonism | Semaglutide | GLP1R | GLP-1 receptor agonist that enhances glucose-dependent insulin secretion; slows gastric emptying; increases pancreatic β-cell proliferation; reduces glucagon release | - Ozempic - Rybelsus - Wegovy |
- Type 2 diabetes mellitus (T2DM) |
| GLP-1 receptor agonism | Dulaglutide | GLP1R | GLP-1 receptor agonist that increases glucose-dependent insulin secretion, decreases glucagon secretion, and slows gastric emptying | - Trulicity | - Type 2 diabetes mellitus - MACE risk reduction in T2DM with CV disease or risk factors |
| HIV integrase and reverse transcriptase inhibition | Bictegravir/Emtricitabine/Tenofovir Alafenamide | HIV-1 integrase; HIV-1 reverse transcriptase | Three-drug regimen: bictegravir is an HIV-1 integrase strand transfer inhibitor (INSTI); emtricitabine (FTC) and tenofovir alafenamide (TAF) are HIV-1 nucleoside analog reverse transcriptase inhibitors (NRTIs) | - Biktarvy | - HIV-1 infection in adults and pediatric patients (≥14 kg) |
| IL-12/IL-23 inhibition | Ustekinumab | IL12B (p40 subunit shared by IL-12 and IL-23) | Human monoclonal antibody that binds the shared p40 subunit of IL-12 and IL-23, preventing their interaction with the IL-12 receptor β1 subunit and thereby inhibiting IL-12/IL-23–mediated signaling and cytokine production | - Stelara - Wezlana - Imuldosa - Otulfi - Pyzchiva - Selarsdi - Steqeyma - Yesintek |
- Plaque psoriasis - Psoriatic arthritis - Crohn's disease - Ulcerative colitis |
| IL-17A inhibition | Secukinumab | IL17A | Selective IL-17A antagonist that binds IL-17A and inhibits interaction with the IL-17 receptor; neutralizes IL-17A activity and inhibits release of proinflammatory cytokines, chemokines, and mediators of tissue damage | - Cosentyx | - Plaque psoriasis - Psoriatic arthritis - Ankylosing spondylitis - Non-radiographic axial spondyloarthritis - Enthesitis-related arthritis - Hidradenitis suppurativa |
| IL-23 inhibition | Risankizumab | IL23A (IL-23 p19 subunit) | Binds the p19 subunit of IL-23 and inhibits IL-23 from interacting with the IL-23 receptor and subsequent signaling | - Skyrizi | - Plaque psoriasis - Psoriatic arthritis - Crohn's disease |
| IL-23 inhibition | Guselkumab | IL-23 (IL23A) / IL-23 receptor (IL23R) | Human IgG1λ monoclonal antibody that binds selectively to IL-23 and inhibits IL-23 bioactivity by blocking IL-23 interaction with the cell-surface IL-23 receptor, disrupting IL-23–mediated signaling, activation, and cytokine cascades | - Tremfya | - Plaque psoriasis - Psoriatic arthritis - Ulcerative colitis - Crohn's disease |
| IL-4/IL-13 pathway inhibition | Dupilumab | IL4R (IL-4 receptor alpha subunit, IL-4Rα) | Binds IL-4Rα to inhibit IL-4 and IL-13 signaling (dual blockade of type 2 inflammatory pathways) | - Dupixent | - Atopic dermatitis - Asthma - Chronic rhinosinusitis with nasal polyposis - Eosinophilic esophagitis - Prurigo nodularis |
| Immunomodulation | Immune Globulin Intravenous (Human) | Fc receptors (FcRs) and complement proteins | Immunomodulating therapy via Fc receptor (FcR) blockade, neutralization of pathogenic autoantibodies (anti-idiotypic effects), and complement cascade inhibition | - PRIVIGEN | - Humoral immunodeficiency |
| JAK inhibition | Upadacitinib | JAK1/JAK3 | Janus kinase (JAK) inhibitor; in human leukocyte cellular assays, inhibited cytokine-induced STAT phosphorylation mediated by JAK1 and JAK1/JAK3 | - Rinvoq | - Moderately to severely active ulcerative colitis - Moderately to severely active Crohn's disease |
| Neprilysin inhibition + RAAS blockade | Sacubitril/Valsartan | Neprilysin (MME) / angiotensin II receptor type 1 (AGTR1) | Neprilysin inhibition plus angiotensin II receptor blockade: sacubitril inhibits neprilysin to prevent breakdown of natriuretic peptides (prolonging favorable effects), while valsartan blocks the RAAS via angiotensin II receptor antagonism | - Entresto | - Chronic heart failure in adults - Symptomatic heart failure with LV systolic dysfunction (pediatric ≥1 year) |
| PD-1 checkpoint inhibition | Pembrolizumab | PDCD1 (PD-1) | Anti–PD-1 checkpoint blockade: binds PD-1 and blocks PD-L1/PD-L2 from interacting with PD-1 to help restore T-cell response and immune response | - Keytruda | - Unresectable or metastatic melanoma - Non-small cell lung cancer (NSCLC) - Urothelial carcinoma - MSI-H or dMMR solid tumors - Additional approved oncology indications |
| RANKL inhibition | Denosumab | TNFSF11 (RANKL) | RANK ligand (RANKL) inhibitor that binds RANKL and competitively inhibits its binding to RANK, inhibiting osteoclast formation, function, and survival and reducing bone resorption | - Prolia | - Postmenopausal osteoporosis at high fracture risk - Osteoporosis in men at high fracture risk - Glucocorticoid-induced osteoporosis at high fracture risk - Bone loss from androgen deprivation therapy (prostate cancer, men) - Bone loss from aromatase inhibitor therapy (breast cancer, women) |
| SARS-CoV-2 mRNA vaccination | Tozinameran | SARS-CoV-2 spike (S) protein antigen | Nucleoside-modified mRNA delivered in lipid nanoparticles to direct transient expression of the SARS-CoV-2 spike (S) antigen, eliciting neutralizing antibody and cellular immune responses | - Comirnaty | - COVID-19 prevention in individuals ≥12 years - COVID-19 prevention in individuals ≥5 years |
| SARS-CoV-2 mRNA vaccination | Elasomeran | SARS-CoV-2 spike (S) protein antigen | Synthetic nucleoside-modified mRNA encapsulated in lipid nanoparticles (LNPs) that delivers instructions for the full-length, pre-fusion stabilized SARS-CoV-2 spike (S) protein, leading to S antigen expression and an immune response that protects against SARS-CoV-2. | - COVID-19 Vaccine Moderna - Spikevax |
- COVID-19 (SARS-CoV-2) prevention |
| T-cell costimulation modulation | Abatacept | CD80 and CD86 (cluster of differentiation 80 and cluster of differentiation 86) | Selective costimulation modulator that inhibits T-cell activation by binding CD80 and CD86, thereby blocking interaction with CD28 | - Orencia | - Rheumatoid arthritis - Juvenile idiopathic arthritis (polyarticular JIA) - Psoriatic arthritis |
| TNF-α inhibition | Adalimumab | TNF | TNF-α neutralization by binding TNF-α and blocking connection to p55/p75 TNF receptors | - Humira | - Rheumatoid arthritis - Juvenile idiopathic arthritis - Psoriatic arthritis - Ankylosing spondylitis - Crohn's disease - Ulcerative colitis - Plaque psoriasis - Hidradenitis suppurativa - Uveitis |
| TNF-α inhibition | Etanercept | TNF | Dimeric soluble form of the p75 TNF receptor (fusion protein) that binds TNF and sequesters it from circulation, thereby modulating TNF-regulated inflammatory processes | - Benepali - Enbrel - Erelzi - Eticovo - Nepexto |
- Rheumatoid arthritis - Polyarticular juvenile idiopathic arthritis - Psoriatic arthritis - Ankylosing spondylitis - Plaque psoriasis - Juvenile psoriatic arthritis |
| VEGF inhibition | Aflibercept | VEGFR-1 (FLT1) / VEGFR-2 (KDR) | Soluble decoy receptor that binds VEGF-A and PlGF, inhibiting binding/activation of cognate VEGF receptors and thereby reducing neovascularization and vascular permeability | - Eylea | - Neovascular (wet) age-related macular degeneration (AMD) - Macular edema following retinal vein occlusion (RVO) - Diabetic macular edema (DME) - Diabetic retinopathy (DR) |
| VZV immunity activation | Zoster Vaccine Recombinant Adjuvanted | Varicella-zoster virus glycoprotein E (gE) antigen | Recombinant VZV glycoprotein E (gE) subunit antigen combined with the AS01B adjuvant system to drive immune responses against VZV | - Shingrix | - Herpes zoster (shingles) prevention in immunocompromised adults (≥18 years) |