Options mapped
Paroxysmal nocturnal hemoglobinuria (PNH): options by country
Sourced options by country plus visit-prep questions for Paroxysmal nocturnal hemoglobinuria (PNH). Each line links to its regulator, HTA, or guideline source. This page maps options; it does not recommend or rank them.
Options mappedRare diseaseLast checked June 2026
What this page does
Maps options by country
It maps sourced options by country alongside diagnosis wording, stage, test results, specialists, and trial-search terms.
What it does not do
Does not choose treatment
It does not rank treatments, recommend a choice, or decide clinical fit.
Where it comes from
Built on trusted sources
Every option links to a trusted regulator, HTA, or guideline source, and the list grows as new sources pass verification.
Information to gather before the next visit
- Is the PNH issue intravascular hemolysis, residual extravascular hemolysis, thrombosis risk, transfusion need, or marrow failure overlap?
- Is the option a C5 inhibitor, C3 inhibitor, factor B inhibitor, factor D add-on, or switch from an existing complement inhibitor?
- What vaccination, REMS, infection-risk, and local payer requirements apply?
- Is the person already stable on a C5 inhibitor, complement-inhibitor naive, or experiencing residual EVH?
Trial-search terms to discuss
Options by country
Treatments by country
Regulatory and access status by country, from official sources. It shows what exists and where — not a recommendation.
United States
- eculizumab (Soliris); ravulizumab (Ultomiris); pegcetacoplan (Empaveli); iptacopan (Fabhalta); crovalimab-akkz (Piasky); danicopan (Voydeya)[1]FDA-approvedPNH clone / complement-mediated hemolysis; extravascular hemolysis subset when specified; Complement inhibition for PNH hemolysis; proximal complement inhibition in adults; Voydeya specifically as add-on for extravascular hemolysis while on ravulizumab or eculizumab. · Complement inhibitors carry serious infection/vaccination/REMS warnings in the labels. PNH indication scopes vary by age, weight, monotherapy versus add-on status, and intravascular versus extravascular hemolysis. No sequencing or superiority claim is made. Confidence/conflicts: High for U.S. label claims; no conflict identified.
- eculizumab (Soliris); ravulizumab (Ultomiris); pegcetacoplan (Empaveli); iptacopan (Fabhalta); crovalimab-akkz (Piasky); danicopan (Voydeya)[1]FDA-approvedPNH clone / complement-mediated hemolysis; extravascular hemolysis subset when specified; Complement inhibition for PNH hemolysis; proximal complement inhibition in adults; Voydeya specifically as add-on for extravascular hemolysis while on ravulizumab or eculizumab. · Complement inhibitors carry serious infection/vaccination/REMS warnings in the labels. PNH indication scopes vary by age, weight, monotherapy versus add-on status, and intravascular versus extravascular hemolysis. No sequencing or superiority claim is made. Confidence/conflicts: High for U.S. label claims; no conflict identified.
European Union
- crovalimab (Piasky); danicopan (Voydeya)[2]EMA authorisedcomplement-mediated hemolysis; residual hemolytic anemia/extravascular hemolysis when specified; Piasky C5 inhibitor treatment in PNH; Voydeya add-on for residual hemolytic anemia/EVH while on eculizumab or ravulizumab; China crovalimab for complement-inhibitor-naive adults/adolescents 12+ per Chugai. · China and Japan Piasky/Voydeya claims are manufacturer/regulatory-announcement sourced except EMA Piasky. EU Voydeya source is AstraZeneca announcement, not EMA EPAR in this pass. National reimbursement is not established. Confidence/conflicts: High for EMA Piasky; medium for Japan/China/EU Voydeya and China/Japan Piasky until direct PMDA/NMPA/EMA product pages are fetched.
- crovalimab (Piasky); danicopan (Voydeya)[2]EMA authorisedcomplement-mediated hemolysis; residual hemolytic anemia/extravascular hemolysis when specified; Piasky C5 inhibitor treatment in PNH; Voydeya add-on for residual hemolytic anemia/EVH while on eculizumab or ravulizumab; China crovalimab for complement-inhibitor-naive adults/adolescents 12+ per Chugai. · China and Japan Piasky/Voydeya claims are manufacturer/regulatory-announcement sourced except EMA Piasky. EU Voydeya source is AstraZeneca announcement, not EMA EPAR in this pass. National reimbursement is not established. Confidence/conflicts: High for EMA Piasky; medium for Japan/China/EU Voydeya and China/Japan Piasky until direct PMDA/NMPA/EMA product pages are fetched.
Australia
- eculizumab (Soliris); ravulizumab (Ultomiris); pegcetacoplan (Empaveli)[3]TGA-registered (Australia)PNH clone / complement-mediated hemolysis; PIGA somatic mutation context described by source; C5 inhibition for PNH hemolysis; ravulizumab for adult/paediatric PNH body weight 10 kg or above in stated settings; pegcetacoplan after inadequate response to or intolerance of a C5 inhibitor. · The AusPAR summarizes Australian regulator status and indication context but is not a payer document. Complement inhibitor infection/vaccination precautions require current product information and local hematology review. Confidence/conflicts: High for Australian PNH approval context for Soliris, Ultomiris, and Empaveli; direct current PI and PBS criteria remain source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.
- iptacopan (Fabhalta)[4]TGA-registered (Australia)PNH clone / complement-mediated hemolysis; adult PNH in Australia. · The TGA AusPAR verifies registration and approved indication but not PBS subsidy or sequencing versus C5 inhibitors or pegcetacoplan. Infection prevention requirements should be checked in the current PI. Confidence/conflicts: High for Australian registration and indication; payer status remains source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.
Thailand
- ravulizumab (Ultomiris)[5]Approvedcomplement-mediated hemolysis context; source defines PNH treatment population by hemolysis with clinical symptoms indicating high disease activity, or clinical stability after eculizumab for at least 6 months; complement-inhibitor-naive PNH with high disease activity symptoms, or switch/maintenance context after at least 6 months of eculizumab stability; adult and pediatric patients weighing at least 10 kg. · Ravulizumab must be administered by a healthcare professional under supervision of a physician experienced in hematological disorders. The label states PNH is chronic and ravulizumab treatment is recommended to continue for the patient's lifetime unless discontinuation is clinically indicated. It also states pediatric PNH patients under 30 kg were not studied and that dosing for those patients is extrapolated from aHUS PK/PD data. Meningococcal vaccination/prophylaxis is described in the clinical-study context; this entry does not establish Thai reimbursement, center availability, or whether another complement inhibitor is preferred. Confidence/conflicts: High for Thai NDI Ultomiris PNH label content; no conflict identified. Thai reimbursement/access and direct Thai marketing-authorization metadata remain unresolved.
Canada
- crovalimab (Piasky)[6]Health Canada approvedPNH clone / complement-mediated hemolysis; PNH in adults and adolescents 13 years and older weighing at least 40 kg in Canada. · The indication does not establish payer coverage, self-administration suitability, or switching criteria. Complement inhibition infection risk and vaccination requirements should be confirmed from the current monograph and care team. Confidence/conflicts: High for Canadian crovalimab indication; reimbursement and access pathway remain source-pending. No conflict identified.
- danicopan (Voydeya) add-on to ravulizumab or eculizumab[7]ApprovedPNH clone; EVH while on terminal complement inhibition; adult PNH with residual hemolytic anemia due to EVH as add-on to ravulizumab or eculizumab. · The monograph states Health Canada has not authorized pediatric use. CDA-AMC conditions include prior C5 inhibitor reimbursement criteria, stable C5 inhibitor treatment, persistent anemia due to EVH, reticulocyte threshold, hematologist prescribing, and price/implementation conditions; provincial adoption remains source-pending. Confidence/conflicts: High for Canadian add-on indication and CDA-AMC conditional recommendation; provincial implementation remains source-pending. No conflict identified.
- iptacopan (Fabhalta)[8]Health Canada approvedPNH clone / complement-mediated hemolysis; adult PNH with hemolytic anemia in Canada. · Health Canada indication is monotherapy and adult-only. The product monograph includes contraindications and serious infection precautions; reimbursement and provincial availability were not established by these sources. Confidence/conflicts: High for Canadian approval and label; reimbursement remains source-pending. No conflict identified.
Sources
- U.S. Food and Drug Administration — official drug label · official drug label
- European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
- Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
- Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
- Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
- Health Canada product monograph repository / Roche Canada — official product monograph · official product monograph
- Alexion Canada / Health Canada product monograph — official product monograph · official product monograph
- Health Canada Drug and Health Products Portal — Summary Basis of Decision · Summary Basis of Decision
This is official regulatory and access status only — not medical advice, not a recommendation, and not a statement about eligibility. Whether any option fits depends on your situation and your oncology team. Status changes over time; confirm the current position with the linked source. Last checked 2026-06-12.
Beyond approved care
In clinical trials & emerging options
Options that are not — or not yet — an approved standard where you live: studies, clinical trials, off-label use, and early evidence that your own oncologist may not raise. Each is labeled by how strong the evidence is. A listing here is information to research and discuss with your team; it does not mean a treatment is proven, safe for you, or available today.
In clinical trials
- pegcetacoplan; danicopan; pozelimab plus cemdisiran; HSK39297; MY008211A; Elizaria; REGN3918Clinical trial · NCT03500549Clinical trialTrial only (registry)Russia · PNH clone / complement-mediated hemolysis; Complement-inhibitor PNH studies, anti-C5/biosimilar/alternative complement pathways, and add-on or comparator studies as stated in registry interventions. · Some studies are completed or terminated. Elizaria records verify Russia study presence, not a current Russian regulator label. China studies are investigational unless supported by separate NMPA sources. Thailand had no direct ClinicalTrials.gov PNH hit in this pass. Confidence/conflicts: High for registry geography/status; low for routine country access except where separate regulator/announcement sources are recorded. ClinicalTrials.gov — clinical-trial registry
- pegcetacoplan; danicopan; pozelimab plus cemdisiran; HSK39297; MY008211A; Elizaria; REGN3918Clinical trial · NCT03500549Clinical trialTrial only (registry)Russia · PNH clone / complement-mediated hemolysis; Complement-inhibitor PNH studies, anti-C5/biosimilar/alternative complement pathways, and add-on or comparator studies as stated in registry interventions. · Some studies are completed or terminated. Elizaria records verify Russia study presence, not a current Russian regulator label. China studies are investigational unless supported by separate NMPA sources. Thailand had no direct ClinicalTrials.gov PNH hit in this pass. Confidence/conflicts: High for registry geography/status; low for routine country access except where separate regulator/announcement sources are recorded. ClinicalTrials.gov — clinical-trial registry
A clinical-trial listing or early report shows an option is being studied — not that it works, that it is safe for any one person, or that a site is enrolling today. Whether any of these fits is a conversation for your oncology team and the trial team. Last checked 2026-06-12.