--- title: "Danicopan | PepTracker Pro" url: https://peptrackerpro.com/peptides/danicopan description: "AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is not used as monotherapy." lang: en --- Skip to main content **Educational information only.** This site does not provide medical advice. Read full disclaimer (https://peptrackerpro.com/disclaimer) **Research-only content.** This page is for educational purposes and does not constitute medical advice. Read full disclaimer → (https://peptrackerpro.com/research-disclaimer) # Danicopan High Evidence AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is not used as monotherapy. Aliases Danicopan +14 more Evidence High Evidence Last Updated 2026-10-11 Reading Time 5 min ## What It Is Danicopan (brand name Voydeya, development code ALXN2040; earlier Achillion code ACH-4471) is a small-molecule drug from AstraZeneca's Alexion rare-disease unit that is a first-in-class, orally available inhibitor of complement factor D, and the first complement medicine designed specifically to control the 'extravascular' side of paroxysmal nocturnal hemoglobinuria (PNH). The complement system is part of innate immunity; in PNH, red blood cells lack the protective surface proteins CD55 and CD59 and are destroyed by complement. The established treatments - the anti-C5 antibodies eculizumab and ravulizumab - block the terminal step and stop intravascular hemolysis (red cells bursting inside blood vessels), but in a minority of patients C3 fragments pile up on surviving red cells and those cells are then cleared by the spleen and liver, a residual 'extravascular hemolysis' (EVH) that leaves patients anemic and sometimes transfusion-dependent despite C5 therapy. Danicopan is added on top of a C5 inhibitor to shut down the alternative-pathway amplification loop upstream, at factor D, reducing the C3-fragment opsonization that drives EVH while the C5 inhibitor continues to control intravascular hemolysis. It is taken by mouth three times a day and was FDA-approved on March 29, 2024 on the strength of the Phase 3 ALPHA trial. Danicopan is a prescription medicine prescribed and monitored by hematologists for a confirmed diagnosis - not a supplement, nootropic, or research chemical. Also known as: Danicopan, Voydeya, danicopan, ALXN2040, ALXN-2040, ACH-4471, ACH-0144471, oral factor D inhibitor, factor D inhibitor, complement factor D inhibitor, alternative complement pathway inhibitor, Voydeya PNH, danicopan add-on, AstraZeneca complement inhibitor, Alexion danicopan ## Why Researchers Study It Danicopan is studied as proof that the complement alternative pathway can be controlled with an oral pill at factor D, and specifically as the first targeted answer to extravascular hemolysis (EVH) - the residual anemia that terminal C5 inhibitors (eculizumab, ravulizumab) cannot fix. In PNH, blocking C5 stops intravascular hemolysis but lets C3 fragments accumulate on surviving red cells, which are then cleared extravascularly; by inhibiting factor D upstream, danicopan reduces that C3-fragment opsonization while the C5 inhibitor keeps terminal complement suppressed. It is a key datapoint in the broader question of where to intervene in the alternative pathway - factor D (danicopan) versus factor B (iptacopan) versus C3 (pegcetacoplan) - and in whether add-on proximal inhibition or a switch to proximal monotherapy is the better route for EVH. Its selective, oral, small-molecule mechanism also makes it a template for extending factor D inhibition to other complement-driven diseases. ## Proposed Mechanisms - Selectively inhibits complement factor D, the serine protease that cleaves factor B to form the alternative-pathway C3 convertase, blocking formation and activity of that convertase - Acts at the proximal amplification loop of the alternative pathway, upstream of C3 cleavage and the terminal C5 step, so it dampens the C3-fragment (C3b/iC3b) opsonization that marks PNH red cells for extravascular clearance - Added on top of a C5 inhibitor (ravulizumab or eculizumab): the C5 inhibitor continues to block terminal complement and intravascular hemolysis, while danicopan reduces the extravascular hemolysis caused by C3-fragment deposition - Oral small molecule dosed three times daily (150 mg, up-titratable to 200 mg), giving proximal alternative-pathway control without an infusion or injection ## Evidence Snapshot High Evidence Low Medium High | Study Type | Model | Outcome | Link | | --- | --- | --- | --- | | Phase 3, PNH with clinically significant extravascular hemolysis (ALPHA) | 86 adults with PNH and clinically significant EVH on stable ravulizumab or eculizumab, randomized 2:1 to add danicopan vs placebo on top of the C5 inhibitor | Mean hemoglobin increase at week 12 of +2.94 g/dL with danicopan vs +0.50 g/dL with placebo (p=0.0001); a 46.9 percentage-point greater proportion achieved a hemoglobin rise of at least 2 g/dL by week 12 without transfusion (p<0.0001); improved fatigue (FACIT-Fatigue); no meningococcal infections, hemolysis-related discontinuations, or deaths - basis for FDA approval on March 29, 2024 | Source: https://www.ncbi.nlm.nih.gov/books/NBK613820/ | | Regulatory milestone - FDA approval | Add-on therapy to ravulizumab or eculizumab in adults with PNH and clinically significant extravascular hemolysis; not indicated as monotherapy | FDA approved Voydeya (danicopan) on March 29, 2024 as the first oral factor D inhibitor for extravascular hemolysis in PNH; also approved in the EU and Japan in 2024; dispensed through a REMS with a boxed warning for serious encapsulated-bacterial infections | Source: https://www.drugs.com/history/voydeya.html | ## Commonly Discussed Benefits immunology: https://peptrackerpro.com/benefits/immunology rare-disease: https://peptrackerpro.com/benefits/rare-disease complement-inhibitor: https://peptrackerpro.com/benefits/complement-inhibitor Hematology: https://peptrackerpro.com/benefits/hematology anemia: https://peptrackerpro.com/benefits/anemia Researching Danicopan? Track it, set reminders, and keep notes in the free app. Track in App (https://app.peptrackerpro.com/?add=danicopan) ## Safety & Cautions - Carries a boxed warning for serious and potentially life-threatening infections from encapsulated bacteria (Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae type b) because it suppresses part of the complement immune defense - Dispensed only through a Risk Evaluation and Mitigation Strategy (REMS) program; vaccination against meningococcal and pneumococcal bacteria should be completed or updated at least 2 weeks before the first dose (with antibiotic prophylaxis if treatment cannot wait), and vaccination does not eliminate the infection risk - Initiation is contraindicated in patients with an unresolved serious infection caused by encapsulated bacteria - Is only add-on therapy to ravulizumab or eculizumab and is not effective or indicated as monotherapy; stopping danicopan requires monitoring for a return of hemolysis and PNH symptoms - Can raise liver enzymes (hepatic enzyme increases), so liver tests should be checked before and periodically during treatment - Raises total and LDL cholesterol (hyperlipidemia), so lipids should be monitored and treated if needed - A prescription medicine for a confirmed PNH diagnosis under hematology care - not a supplement, nootropic, or research chemical; any 'danicopan' or 'Voydeya' sold outside a pharmacy is unverified - Common side effects include headache, nausea, vomiting, fever, elevated ALT, high blood pressure, arthralgia, diarrhea, and pain in an extremity ## Comparisons See how Danicopan compares to related peptides: Danicopan vs Pegcetacoplan: https://peptrackerpro.com/compare/danicopan-vs-pegcetacoplan Danicopan vs Cemdisiran: https://peptrackerpro.com/compare/danicopan-vs-cemdisiran Danicopan vs Pozelimab: https://peptrackerpro.com/compare/danicopan-vs-pozelimab Danicopan vs Crovalimab (Piasky): https://peptrackerpro.com/compare/danicopan-vs-crovalimab Danicopan vs Iptacopan: https://peptrackerpro.com/compare/danicopan-vs-iptacopan Danicopan vs Avacopan: https://peptrackerpro.com/compare/danicopan-vs-avacopan ## Calculator Tools Use our research tools to explore dosing and reconstitution data: Reconstitution Calculator: https://peptrackerpro.com/calculators ## Citations 1. [1] FDA label - VOYDEYA (danicopan) tablets, for oral use, prescribing information (2024) PubMed (https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218037s000lbl.pdf) 2. [2] Voydeya (danicopan) FDA Approval History - Drugs.com PubMed (https://www.drugs.com/history/voydeya.html) 3. [3] Factor D Inhibitor Danicopan Approved as Add-On Therapy for PNH - AJMC PubMed (https://www.ajmc.com/view/factor-d-inhibitor-danicopan-approved-as-add-on-therapy-for-pnh) 4. [4] FDA Approves Danicopan as Add-On Therapy for Extravascular Hemolysis in PNH - OncLive PubMed (https://www.onclive.com/view/fda-approves-danicopan-as-add-on-therapy-for-extravascular-hemolysis-in-pnh) 5. [5] Clinical Review - Danicopan (Voydeya) - NCBI Bookshelf / CADTH PubMed (https://www.ncbi.nlm.nih.gov/books/NBK613820/) 6. [6] Danicopan Approved as Add-On Therapy to Ravulizumab or Eculizumab for EVH in Adults With PNH - The ASCO Post PubMed (https://ascopost.com/news/april-2024/danicopan-approved-as-add-on-therapy-to-ravulizumab-or-eculizumab-for-extravascular-hemolysis-in-adults-with-paroxysmal-nocturnal-hemoglobinuria/) ### Keep researching in the app - Log Danicopan to your private tracker - Set a dosing reminder - Compare it side-by-side with your stack Open PepTracker Pro Free: https://app.peptrackerpro.com/?add=danicopan Browse more peptides: https://peptrackerpro.com/peptides ## Related Peptides ### Pegcetacoplan High Evidence A PEGylated cyclic-peptide inhibitor of complement component 3 (C3) built from two compstatin (Cp05) peptide domains bridged by a 40-kDa PEG chain; marketed as Empaveli/Aspaveli (subcutaneous, for paroxysmal nocturnal hemoglobinuria and, since July 2025, C3 glomerulopathy and primary IC-MPGN) and Syfovre (intravitreal, for geographic atrophy in age-related macular degeneration). It is one of the few complement-targeting therapeutic peptides to reach the market. Immune Support: https://peptrackerpro.com/benefits/immune-support Inflammation: https://peptrackerpro.com/benefits/inflammation View Details: https://peptrackerpro.com/peptides/pegcetacoplan \+ Compare: https://peptrackerpro.com/compare?select=pegcetacoplan Track in App: https://app.peptrackerpro.com/?add=pegcetacoplan ### Cemdisiran High Evidence Cemdisiran is an investigational GalNAc-conjugated small interfering RNA (siRNA) that silences the gene for complement component 5 (C5) in the liver, durably lowering circulating C5. It is being developed by Regeneron (under a license from Alnylam, which originated the molecule as ALN-CC5) as a subcutaneous, once-every-12-weeks treatment for adults with anti-acetylcholine-receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) - an autoimmune disease in which antibodies and complement attack the neuromuscular junction, so that activation of C5 and formation of the membrane attack complex destroy the acetylcholine receptors that make muscles contract, causing fluctuating, fatigable weakness. By knocking down C5 production rather than blocking the circulating protein with an antibody or peptide, cemdisiran offers quarterly dosing instead of the weekly-to-monthly injections of existing complement inhibitors. The pivotal Phase 3 NIMBLE trial (NCT05070858) - a randomized, double-blind, placebo-controlled study of about 284 adults with gMG, published in The Lancet in 2026 and presented at the American Academy of Neurology annual meeting - compared cemdisiran 600 mg given subcutaneously every 12 weeks against placebo and against a pozelimab-plus-cemdisiran combination. In the cemdisiran monotherapy arm the mean MG-ADL (Myasthenia Gravis Activities of Daily Living) score improved by 4.5 points from baseline at week 24 versus 2.2 points on placebo, a placebo-adjusted improvement of 2.3 points (p<0.001), with 76.6% of cemdisiran patients achieving at least a 3-point MG-ADL improvement versus 44.1% on placebo; the QMG (Quantitative Myasthenia Gravis) score improved by 4.2 versus 1.5 points, a placebo-adjusted 2.8 points (p=0.002), and benefits appeared within two weeks. No serious infections, meningococcal infections or deaths occurred during the double-blind treatment period, and there were no discontinuations for adverse events through week 24. Regeneron submitted a regulatory application in the first quarter of 2026; the FDA accepted it under Priority Review with a target action date in November 2026, and a European application is under review with a decision expected in the second half of 2027. If approved, cemdisiran would be the first RNA-interference (siRNA) therapy for generalized myasthenia gravis. It is investigational and not yet approved for this use anywhere. immunology: https://peptrackerpro.com/benefits/immunology neuromuscular: https://peptrackerpro.com/benefits/neuromuscular myasthenia-gravis: https://peptrackerpro.com/benefits/myasthenia-gravis complement-inhibition: https://peptrackerpro.com/benefits/complement-inhibition View Details: https://peptrackerpro.com/peptides/cemdisiran \+ Compare: https://peptrackerpro.com/compare?select=cemdisiran Track in App: https://app.peptrackerpro.com/?add=cemdisiran ### Pozelimab High Evidence Regeneron's fully human anti-C5 monoclonal antibody (Veopoz) — the first approved treatment for CHAPLE disease and the antibody half of the cemdisiran combination for complement-driven disease. immunology: https://peptrackerpro.com/benefits/immunology rare-disease: https://peptrackerpro.com/benefits/rare-disease complement-inhibition: https://peptrackerpro.com/benefits/complement-inhibition Hematology: https://peptrackerpro.com/benefits/hematology View Details: https://peptrackerpro.com/peptides/pozelimab \+ Compare: https://peptrackerpro.com/compare?select=pozelimab Track in App: https://app.peptrackerpro.com/?add=pozelimab ### Crovalimab (Piasky) High Evidence Chugai/Roche's humanized recycling anti-C5 monoclonal antibody (Piasky) — FDA-approved for paroxysmal nocturnal hemoglobinuria as a low-volume, once-monthly subcutaneous injection patients can give themselves. View Details: https://peptrackerpro.com/peptides/crovalimab \+ Compare: https://peptrackerpro.com/compare?select=crovalimab Track in App: https://app.peptrackerpro.com/?add=crovalimab ### Iptacopan High Evidence Novartis's Fabhalta (iptacopan, development code LNP023) - the first approved oral, first-in-class small-molecule factor B inhibitor that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 200 mg capsule twice daily, it is FDA-approved for paroxysmal nocturnal hemoglobinuria (PNH), for primary IgA nephropathy (IgAN), and as the first-ever treatment for C3 glomerulopathy (C3G), and in July 2026 it became the first and only complement inhibitor to win traditional FDA approval for slowing kidney function decline in IgAN, cutting the annual rate of eGFR loss by about 48% versus placebo in the Phase 3 APPLAUSE-IgAN trial. immunology (https://peptrackerpro.com/benefits/immunology)rare-disease (https://peptrackerpro.com/benefits/rare-disease)complement-inhibitor (https://peptrackerpro.com/benefits/complement-inhibitor)Hematology (https://peptrackerpro.com/benefits/hematology)+1 more View Details: https://peptrackerpro.com/peptides/iptacopan \+ Compare: https://peptrackerpro.com/compare?select=iptacopan Track in App: https://app.peptrackerpro.com/?add=iptacopan ### Avacopan High Evidence Avacopan (brand Tavneos, development code CCX168) is a first-in-class, oral, small-molecule antagonist of the complement C5a receptor (C5aR1, also called CD88) from ChemoCentryx (now part of Amgen). Taken as 30 mg (three 10 mg capsules) twice daily with food, it blocks C5a from activating neutrophils and is FDA-approved as an adjunctive treatment for adults with severe active ANCA-associated vasculitis (granulomatosis with polyangiitis, GPA, and microscopic polyangiitis, MPA) alongside standard therapy (rituximab or cyclophosphamide). It is designed to let patients taper off high-dose glucocorticoids, not to replace immunosuppression. immunology (https://peptrackerpro.com/benefits/immunology)rare-disease (https://peptrackerpro.com/benefits/rare-disease)complement-inhibitor (https://peptrackerpro.com/benefits/complement-inhibitor)vasculitis (https://peptrackerpro.com/benefits/vasculitis)+1 more View Details: https://peptrackerpro.com/peptides/avacopan \+ Compare: https://peptrackerpro.com/compare?select=avacopan Track in App: https://app.peptrackerpro.com/?add=avacopan ## Structured data ```json [ { "@context": "https://schema.org", "@type": "Organization", "name": "PepTracker Pro", "url": "https://peptrackerpro.com", "logo": "https://peptrackerpro.com/favicon.png", "description": "Evidence-based educational resource dedicated to improving peptide research literacy.", "contactPoint": { "@type": "ContactPoint", "email": "hello@peptrackerpro.com", "contactType": "customer support" }, "sameAs": [] }, { "@context": "https://schema.org", "@type": "MedicalWebPage", "name": "Danicopan", "description": "AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). 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AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is not used as monotherapy." } }, { "@type": "Question", "name": "People comparing proximal complement options - factor D (danicopan) versus factor B (iptacopan) versus C3 (pegcetacoplan) - for controlling the extravascular hemolysis that terminal C5 inhibitors leave running?", "acceptedAnswer": { "@type": "Answer", "text": "Danicopan is a research peptide studied in preclinical models. AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is not used as monotherapy." } }, { "@type": "Question", "name": "Clinicians deciding between adding an oral factor D inhibitor on top of existing C5 therapy versus switching the patient to a proximal monotherapy such as iptacopan or pegcetacoplan?", "acceptedAnswer": { "@type": "Answer", "text": "Danicopan is a research peptide studied in preclinical models. AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is not used as monotherapy." } }, { "@type": "Question", "name": "Researchers and students studying the complement alternative pathway and how blocking factor D, factor B, or C3 differs from blocking terminal C5?", "acceptedAnswer": { "@type": "Answer", "text": "Danicopan is a research peptide studied in preclinical models. AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is not used as monotherapy." } }, { "@type": "Question", "name": "Patients weighing the convenience and burden of an oral three-times-daily add-on against staying on a C5 inhibitor alone?", "acceptedAnswer": { "@type": "Answer", "text": "Danicopan is a research peptide studied in preclinical models. AstraZeneca/Alexion's Voydeya (danicopan, development code ALXN2040) - a first-in-class, oral, small-molecule inhibitor of complement factor D that selectively blocks the alternative complement pathway at its proximal amplification loop; taken as a 150 mg tablet three times daily (up-titratable to 200 mg three times daily), it is FDA-approved as add-on therapy to the C5 inhibitors ravulizumab or eculizumab to treat clinically significant extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH). It is not used as monotherapy." } } ] } ] ```