# 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.

## Browse all mechanisms

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<br />                    | - Castration-resistant prostate cancer (CRPC)<br />- Metastatic castration-sensitive prostate cancer<br />- 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)<br />- 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)<br />- 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%)<br />- 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<br />- Zepbound       | - Type 2 diabetes mellitus<br />- Chronic weight management (obesity or overweight with comorbidities)<br />- 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)<br />- DVT prophylaxis after hip or knee replacement<br />- Deep vein thrombosis (DVT) treatment<br />- Pulmonary embolism (PE) treatment<br />- 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)<br />- Deep vein thrombosis (DVT) treatment<br />- Pulmonary embolism (PE) treatment<br />- Recurrent DVT/PE risk reduction<br />- DVT prophylaxis after hip or knee replacement<br />- VTE prophylaxis in acutely ill medical patients<br />- Major CV event risk reduction in CAD<br />- Major thrombotic event risk reduction in PAD<br />- Pediatric VTE treatment and recurrence risk reduction<br />- 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<br />- Rybelsus<br />- 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<br />- 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<br />- Wezlana<br />- Imuldosa<br />- Otulfi<br />- Pyzchiva<br />- Selarsdi<br />- Steqeyma<br />- Yesintek | - Plaque psoriasis<br />- Psoriatic arthritis<br />- Crohn's disease<br />- 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<br />- Psoriatic arthritis<br />- Ankylosing spondylitis<br />- Non-radiographic axial spondyloarthritis<br />- Enthesitis-related arthritis<br />- 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<br />- Psoriatic arthritis<br />- 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<br />- Psoriatic arthritis<br />- Ulcerative colitis<br />- 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<br />- Asthma<br />- Chronic rhinosinusitis with nasal polyposis<br />- Eosinophilic esophagitis<br />- 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<br />- 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<br />- 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<br />- Non-small cell lung cancer (NSCLC)<br />- Urothelial carcinoma<br />- MSI-H or dMMR solid tumors<br />- 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<br />- Osteoporosis in men at high fracture risk<br />- Glucocorticoid-induced osteoporosis at high fracture risk<br />- Bone loss from androgen deprivation therapy (prostate cancer, men)<br />- 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<br />- 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<br />- 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<br />- Juvenile idiopathic arthritis (polyarticular JIA)<br />- 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<br />- Juvenile idiopathic arthritis<br />- Psoriatic arthritis<br />- Ankylosing spondylitis<br />- Crohn's disease<br />- Ulcerative colitis<br />- Plaque psoriasis<br />- Hidradenitis suppurativa<br />- 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<br />- Enbrel<br />- Erelzi<br />- Eticovo<br />- Nepexto | - Rheumatoid arthritis<br />- Polyarticular juvenile idiopathic arthritis<br />- Psoriatic arthritis<br />- Ankylosing spondylitis<br />- Plaque psoriasis<br />- 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)<br />- Macular edema following retinal vein occlusion (RVO)<br />- Diabetic macular edema (DME)<br />- 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)                  |