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Paroxysmal Nocturnal Hemoglobinuria

26
Pipeline Programs
18
Companies
50
Clinical Trials
5 recruiting
4
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
4
0
6
0
10
6
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
12100%
+ 38 programs with unclassified modality

On Market (4)

Approved therapies currently available

AstraZeneca
SOLIRISApproved
eculizumab
AstraZeneca
Complement Inhibitor [EPC]iv (infusion)2007
223M Part D
Roche
PIASKYApproved
crovalimab
Roche
Complement C5 Inhibitor [EPC]intravenous2024
Regeneron
VEOPOZApproved
pozelimab
Regeneron
Complement Inhibitor [EPC]injection2023
AstraZeneca
VOYDEYAApproved
danicopan
AstraZeneca
Complement Factor D Inhibitor [EPC]oral2024

Competitive Landscape

18 companies ranked by most advanced pipeline stage

AstraZeneca
AstraZenecaCAMBRIDGE, United Kingdom
6 programs
1
1
SOLIRIS(eculizumab)APPROVED_FOR_MARKETINGMonoclonal Antibody5 trials
ACH-0144471Phase 21 trial
VOYDEYA(danicopan)AVAILABLE5 trials
Paroxysmal Nocturnal Hemoglobinuria (PNH) RegistryN/A1 trial
Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies StudyN/A1 trial
+1 more programs
Active Trials
NCT04802083No Longer Available
NCT00438789Approved For Marketing
NCT04355494No Longer Available
+11 more trials
Regeneron
RegeneronTARRYTOWN, NY
5 programs
1
VEOPOZ(Pozelimab)PHASE_2Monoclonal Antibody5 trials
PozelimabPHASE_2Monoclonal Antibody1 trial
PozelimabPHASE_3Monoclonal Antibody1 trial
PozelimabPHASE_3Monoclonal Antibody1 trial
REGN3918PHASE_32 trials
Active Trials
NCT06028594No Longer Available
NCT07142343Recruiting5Est. Feb 2030
NCT05131204Terminated3Est. Jul 2023
+7 more trials
Roche
RocheSTAVANGER NORWAY, Norway
2 programs
1
CrovalimabPHASE_3Monoclonal Antibody
CrovalimabPHASE_3Monoclonal Antibody
UNION therapeutics
UNION therapeuticsDenmark - Hellerup
1 program
1
sirolimusPhase 41 trial
Active Trials
NCT03866681Unknown40Est. Apr 2022
Sandoz
SandozAustria - Kundl
8 programs
2
2
IptacopanPhase 3
IptacopanPhase 3
LFG316Phase 2
LNP023Phase 2
LNP023N/A
+3 more programs
Createrna Science
Createrna ScienceChina - Shanghai
6 programs
4
1
1
MY008211A tabletsPhase 31 trial
MY008211A tabletsPhase 21 trial
MY008211A tabletsPhase 11 trial
MY008211A tabletsPhase 11 trial
MY008211A tabletsPhase 11 trial
+1 more programs
Active Trials
NCT06543459Not Yet Recruiting16Est. Dec 2024
NCT05642585Completed63Est. Jun 2023
NCT05828472Completed40Est. Jun 2023
+3 more trials
Genentech
GenentechCA - Oceanside
3 programs
3
CrovalimabPhase 3Monoclonal Antibody1 trial
CrovalimabPhase 3Monoclonal Antibody1 trial
CrovalimabPhase 3Monoclonal Antibody1 trial
Active Trials
NCT04432584Active Not Recruiting190Est. Sep 2027
NCT04654468Active Not Recruiting51Est. Dec 2026
NCT04434092Active Not Recruiting210Est. Sep 2027
Akari Therapeutics
2 programs
1
1
rVA576Phase 35 trials
coversinPhase 22 trials
Active Trials
NCT03427060Completed1Est. Feb 2021
NCT02591862Completed1Est. Mar 2018
NCT05061771Withdrawn0Est. Aug 2022
+4 more trials
Amgen
AmgenTHOUSAND OAKS, CA
1 program
1
ABP 959Phase 31 trial
Active Trials
NCT03818607Completed42Est. Jul 2022
Biocad
BiocadRussia - St. Petersburg
1 program
1
BCD-148Phase 31 trial
Active Trials
NCT04060264Completed28Est. Dec 2020
Samsung Bioepis
Samsung BioepisKorea - Incheon
1 program
1
SB12Phase 31 trial
Active Trials
NCT04058158Completed50Est. Oct 2021
Longbio Pharma
Longbio PharmaChina - Shanghai
1 program
1
LP-005 InjectionPhase 21 trial
Active Trials
NCT07266155Not Yet Recruiting30Est. May 2028
BioCryst Pharmaceuticals
4 programs
BCX10013PHASE_11 trial
BCX9930PHASE_1_21 trial
BCX9930PHASE_21 trial
BCX9930PHASE_21 trial
Active Trials
NCT06100900Completed8Est. Dec 2024
NCT04330534Completed168Est. Jan 2021
NCT05741346Terminated28Est. Jan 2025
+1 more trials
Novartis
NovartisBASEL, Switzerland
3 programs
PNH-relevant therapiesN/A1 trial
Specified Drug-use Surveillance of Fabhalta CapsulesN/A1 trial
LFG316PHASE_25 trials
Active Trials
NCT06411626Recruiting128Est. Jun 2026
NCT06606314Recruiting100Est. Sep 2028
NCT02878616Completed8Est. Jul 2017
+4 more trials
NovelMed Therapeutics
NovelMed TherapeuticsOH - Cleveland
2 programs
NM8074PHASE_21 trial
NM8074PHASE_21 trial
Active Trials
NCT05646563Not Yet Recruiting12Est. Aug 2030
NCT05646524Not Yet Recruiting12Est. Sep 2028
Chugai Pharma
Chugai PharmaJapan - Tokyo
2 programs
CrovalimabPHASE_3Monoclonal Antibody
CrovalimabPHASE_3Monoclonal Antibody
IQVIA
IQVIADURHAM, NC
1 program
PegcetacoplanN/A
Swedish Orphan Biovitrum
1 program
PegcetacoplanN/A1 trial
Active Trials
NCT05776472Recruiting200Est. Aug 2029

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
RegeneronPozelimab
AstraZenecaeculizumab
UNION therapeuticssirolimus
AstraZenecaeculizumab
RegeneronPozelimab
AstraZenecaeculizumab
AstraZenecaeculizumab
AstraZenecaeculizumab
Createrna ScienceMY008211A tablets
AstraZenecaeculizumab
AstraZenecaeculizumab
RegeneronPozelimab
AstraZenecadanicopan
RegeneronPozelimab
RegeneronPozelimab

Showing 15 of 50 trials with date data

Clinical Trials (50)

Total enrollment: 2,430 patients across 50 trials

NCT07142343RegeneronPozelimab

A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease

Start: Mar 2026Est. completion: Feb 20305 patients
Phase 4Recruiting
NCT04320602AstraZenecaeculizumab

Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab

Start: Apr 2021Est. completion: Dec 202218 patients
Phase 4Completed
NCT03866681UNION therapeuticssirolimus

Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH

Start: Apr 2019Est. completion: Apr 202240 patients
Phase 4Unknown
NCT00867932AstraZenecaeculizumab

Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start: Oct 2009Est. completion: May 20117 patients
Phase 4Completed
NCT07154745RegeneronPozelimab

A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently

Start: Mar 2026Est. completion: Sep 203135 patients
Phase 3Recruiting
NCT06449001AstraZenecaeculizumab

Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis

Start: Aug 2025Est. completion: Mar 20286 patients
Phase 3Recruiting
NCT06724809AstraZenecaeculizumab

Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD

Start: Jan 2025Est. completion: Dec 202621 patients
Phase 3Active Not Recruiting
NCT06764160AstraZenecaeculizumab

Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Eculizumab in Chinese Adults With gMG

Start: Jan 2025Est. completion: Dec 202515 patients
Phase 3Completed
NCT06932471Createrna ScienceMY008211A tablets

Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment

Start: Aug 2024Est. completion: Dec 202520 patients
Phase 3Recruiting
NCT05876351AstraZenecaeculizumab

Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China

Start: Jul 2023Est. completion: May 202525 patients
Phase 3Completed
NCT05886244AstraZenecaeculizumab

Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China

Start: Jul 2023Est. completion: Apr 202525 patients
Phase 3Completed
NCT05744921RegeneronPozelimab

A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works

Start: Mar 2023Est. completion: Feb 2029202 patients
Phase 3Recruiting
NCT05389449AstraZenecadanicopan

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

Start: Oct 2022Est. completion: Jul 202680 patients
Phase 3Active Not Recruiting
NCT05131204RegeneronPozelimab

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

Start: Oct 2022Est. completion: Jul 20233 patients
Phase 3Terminated
NCT05133531RegeneronPozelimab

A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

Start: Aug 2022Est. completion: Oct 2026202 patients
Phase 3Active Not Recruiting
NCT05061771Akari TherapeuticsrVA576

Nomacopan Therapy in Adult Patients With Bullous Pemphigoid Receiving Adjunct Oral Corticosteroid Therapy (ARREST-BP)

Start: May 2022Est. completion: Aug 20220
Phase 3Withdrawn
NCT04654468GenentechCrovalimab

A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition

Start: Mar 2021Est. completion: Dec 202651 patients
Phase 3Active Not Recruiting
NCT04784455Akari TherapeuticsrVA576

Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

Start: Feb 2021Est. completion: May 202410 patients
Phase 3Terminated
NCT04434092GenentechCrovalimab

A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors

Start: Oct 2020Est. completion: Sep 2027210 patients
Phase 3Active Not Recruiting
NCT04432584GenentechCrovalimab

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

Start: Sep 2020Est. completion: Sep 2027190 patients
Phase 3Active Not Recruiting
NCT04162470RegeneronREGN3918

REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability.

Start: Dec 2019Est. completion: Apr 202224 patients
Phase 3Terminated
NCT04058158Samsung BioepisSB12

A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria

Start: Aug 2019Est. completion: Oct 202150 patients
Phase 3Completed
NCT04060264BiocadBCD-148

Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria

Start: Apr 2019Est. completion: Dec 202028 patients
Phase 3Completed
NCT03748823AstraZenecaeculizumab

Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Start: Feb 2019Est. completion: Aug 2023139 patients
Phase 3Completed
NCT03818607AmgenABP 959

A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH

Start: Jan 2019Est. completion: Jul 202242 patients
Phase 3Completed
NCT03759366AstraZenecaeculizumab

A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)

Start: Dec 2018Est. completion: Nov 202312 patients
Phase 3Completed
NCT03588026Akari TherapeuticsrVA576

Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576

Start: Jun 2018Est. completion: Sep 20209 patients
Phase 3Completed
NCT03829449Akari TherapeuticsrVA576

rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Start: Mar 2017Est. completion: Aug 202015 patients
Phase 3Terminated
NCT02003144AstraZenecaeculizumab

An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients

Start: Jan 2015Est. completion: Jul 2021119 patients
Phase 3Completed
NCT02013037AstraZenecaeculizumab

The De-novo Use of Eculizumab in Presensitized Patients Receiving Cardiac Transplantation

Start: Nov 2012Est. completion: Apr 202036 patients
Phase 3Completed
NCT00122317AstraZenecaeculizumab

Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start: May 2005Est. completion: Oct 2008187 patients
Phase 3Completed
NCT00122304AstraZenecaeculizumab

Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

Start: Dec 2004Est. completion: Nov 200685 patients
Phase 3Completed
NCT00122330AstraZenecaeculizumab

Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Start: Oct 2004Est. completion: Jan 200675 patients
Phase 3Completed
NCT04155424AstraZenecaeculizumab

A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder

Start: Jan 2020Est. completion: Jul 20235 patients
Phase 2/3Terminated
NCT01410916AstraZenecaeculizumab

Safety and Efficacy Study of Eculizumab in Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)

Start: Jul 2011Est. completion: Jun 2012198 patients
Phase 2/3Completed
NCT05646563NovelMed TherapeuticsNM8074

Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris

Start: Jan 2027Est. completion: Aug 203012 patients
Phase 2Not Yet Recruiting
NCT05646524NovelMed TherapeuticsNM8074

Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

Start: Apr 2026Est. completion: Sep 202812 patients
Phase 2Not Yet Recruiting
NCT07266155Longbio PharmaLP-005 Injection

Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start: Dec 2025Est. completion: May 202830 patients
Phase 2Not Yet Recruiting
NCT06050226Createrna ScienceMY008211A tablets

A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start: Jul 2023Est. completion: Nov 202434 patients
Phase 2Completed
NCT05741346BioCryst PharmaceuticalsBCX9930

Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria

Start: Jan 2023Est. completion: Jan 202528 patients
Phase 2Terminated
NCT04725812AstraZenecaeculizumab

Complement Regulation to Undo Systemic Harm in Preeclampsia

Start: Sep 2021Est. completion: Dec 20212 patients
Phase 2Terminated
NCT04811716RegeneronPozelimab

Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy

Start: Jul 2021Est. completion: Oct 202324 patients
Phase 2Completed
NCT04888507RegeneronPozelimab

Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Start: Jul 2021Est. completion: May 20236 patients
Phase 2Completed
NCT04702568BioCryst PharmaceuticalsBCX9930

A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start: Dec 2020Est. completion: Oct 202319 patients
Phase 2Terminated
NCT03946748RegeneronREGN3918

Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start: May 2019Est. completion: Jun 202124 patients
Phase 2Completed
NCT04035733Akari TherapeuticsrVA576

rVA576 in Adult Mild to Moderate Bullous Pemphigoid Subjects

Start: Sep 2018Est. completion: Apr 20209 patients
Phase 2Completed
NCT03459443AstraZenecadanicopan

A Proof of Concept Study for a 12 Month Treatment in Patients With C3G or IC-MPGN Treated With ACH-0144471

Start: Jun 2018Est. completion: Mar 202122 patients
Phase 2Terminated
NCT03427060Akari Therapeuticscoversin

Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

Start: May 2018Est. completion: Feb 20211 patients
Phase 2Completed
NCT03472885AstraZenecaeculizumab

Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Response to Eculizumab

Start: May 2018Est. completion: Jan 202312 patients
Phase 2Completed
NCT03124368AstraZenecadanicopan

A Proof-of-Mechanism Study to Determine the Effect of Danicopan on C3 Levels in Participants With C3G or IC-MPGN

Start: Aug 2017Est. completion: Jan 20196 patients
Phase 2Completed

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

10 late-stage (Phase 3) programs, potential near-term approvals
5 actively recruiting trials targeting 2,430 patients
18 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.