Hemoglobinuria, Paroxysmal

1
Pipeline Programs
1
Companies
23
Clinical Trials
12 recruiting
1
Approved Products

Pipeline by Development Stage

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

On Market (1)

Approved therapies currently available

Novartis
FABHALTAApproved
iptacopan
Novartis
oral2023

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Novartis
NovartisBASEL, Switzerland
1 program
1
FABHALTA(Iptacopan)N/A5 trials
Active Trials
NCT05222412Available
NCT07416162Not Yet Recruiting21Est. Feb 2029
NCT07229235Not Yet Recruiting50Est. Aug 2028
+2 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan
NovartisIptacopan

Showing 15 of 22 trials with date data

Clinical Trials (23)

Total enrollment: 2,558 patients across 23 trials

Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN

Start: Nov 2025Est. completion: Jun 203031 patients
Phase 3Recruiting

A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.

Start: Jul 2024Est. completion: May 2032146 patients
Phase 3Recruiting

Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS

Start: May 2024Est. completion: Jan 2033125 patients
Phase 3Recruiting

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Start: Feb 2024Est. completion: Jul 202950 patients
Phase 3Recruiting

Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN

Start: Oct 2023Est. completion: May 2029106 patients
Phase 3Recruiting

Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.

Start: Apr 2023Est. completion: Oct 202452 patients
Phase 3Completed

Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Start: Jan 2022Est. completion: Aug 202634 patients
Phase 3Active Not Recruiting

A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy

Start: Sep 2021Est. completion: Oct 2032540 patients
Phase 3Recruiting

Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

Start: Jul 2021Est. completion: Jan 202798 patients
Phase 3Recruiting

Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria

Start: Jul 2021Est. completion: Oct 2027208 patients
Phase 3Active Not Recruiting

Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN

Start: Feb 2025Est. completion: Nov 202720 patients
Phase 2Recruiting

Iptacopan in Patients With ANCA Associated Vasculitis

Start: Aug 2024Est. completion: Dec 202684 patients
Phase 2Active Not Recruiting

Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V

Start: Aug 2022Est. completion: Sep 2028240 patients
Phase 2Recruiting

Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

Start: Dec 2021Est. completion: May 202419 patients
Phase 2Terminated

Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Start: Apr 2018Est. completion: Feb 202216 patients
Phase 2Completed

Pharmacokinetic (PK) and Safety Study of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to Matched Control Healthy Participants With Normal Hepatic Function.

Start: Nov 2021Est. completion: Jun 202238 patients
Phase 1Completed

Managed Access Programs for LNP023, Iptacopan

N/AAvailable

A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy

Start: Jul 2026Est. completion: Feb 202921 patients
N/ANot Yet Recruiting

REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria

Start: Dec 2025Est. completion: Aug 202850 patients
N/ANot Yet Recruiting

NIS PDC the Efficacy and Safety of Iptacopan in Adults in Routine Clinical Practice

Start: Jul 2025Est. completion: Jan 2028100 patients
N/ARecruiting

A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China

Start: Jun 2025Est. completion: Apr 202780 patients
N/ARecruiting

Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry

Start: Mar 2025Est. completion: Oct 2029200 patients
N/AActive Not Recruiting

A Mobile App-Based Study to Evaluate Disease Burden and Treatment Patterns in Immunoglobulin A Nephropathy (IgAN) in the US

Start: Mar 2025Est. completion: Jun 2025300 patients
N/ARecruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

12 actively recruiting trials targeting 2,558 patients
1 companies competing in this space