Fabhalta

(Iptacopan)

ApprovedSmall molecule

Iptacopan is used to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH), where the immune system attacks and destroys red blood cells, causing fatigue and other serious complications.[2] Fabhalta is currently approved and in use across 19 indications[4].[1]

Fabhalta is one of 233 small molecule programs targeting Hemolytic Anemia. It is currently in Approved.

Indications

Total Trials

Active Trials

232

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Hemolytic AnemiaActive, not recruiting

Atypical Hemolytic-Uremic SyndromeApproved

VasculitisActive, not recruiting

GlomerulonephritisApproved

IGA GlomerulonephritisApproved

Complement 3 GlomerulopathyApproved

Abnormality of the KidneyActive, not recruiting

Membranoproliferative GlomerulonephritisApproved

Dense Deposit DiseaseApproved

Thrombotic MicroangiopathyApproved

Hemolytic AnemiaPreclinical

Active, not recruiting

Atypical Hemolytic-Uremic SyndromeApproved

Approved

VasculitisPhase 2

Active, not recruiting

GlomerulonephritisApproved

Approved

IGA GlomerulonephritisApproved

Approved

Complement 3 GlomerulopathyApproved

Approved

Abnormality of the KidneyPreclinical

Active, not recruiting

Membranoproliferative GlomerulonephritisApproved

Approved

Dense Deposit DiseaseApproved

Approved

Thrombotic MicroangiopathyApproved

Approved

Showing 10 of 19 indications

Competition

Competitive Landscape

237 competitors in Age-Related Macular Degeneration

#DrugCompanyPhaseTrials

Lucentis

Roche

Phase 3

64 2

Eylea

Regeneron

Phase 3

44 3

Vabysmo

Roche

Phase 3

13 4

Beovu

Novartis

Phase 3

10 5

Fabhalta

Novartis

Approved

1 6

Macugen

Pfizer

Phase 3

6 7

Avastin

BBio-Thera Solutions

Phase 3

18 8

Retaane

AAlcon Research

Phase 3

5 9

Kenalog

BBausch Health Americas, Inc.

Phase 3

2 10

Forzinity

SStealth BioTherapeutics Inc.

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2026

Phase 3Active, not recruiting

Atypical Hemolytic-Uremic Syndrome

NCT04889430

Q4 2026

Phase 2Active, not recruiting

Age-Related Macular Degeneration

NCT05230537

Phase 2Active, not recruiting

Vasculitis

NCT06388941

Q1 2027

Phase 3Recruiting

C3G

NCT04817618

Q4 2027

Phase 3Active, not recruiting

Paroxysmal Nocturnal Hemoglobinuria

NCT04747613

Phase 2Recruiting

Kidney

NCT06797518

Clinical

Trials by Indication[1]

Atypical Hemolytic-Uremic Syndrome2 trials

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

Recruiting· Novartis logo

Novartis·Phase 3·NCT05935215·50 enrolled

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

Active, not recruiting· Novartis logo

Novartis·Phase 3·NCT04889430·34 enrolled

Paroxysmal Nocturnal Hemoglobinuria2 trials

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

Completed· Novartis logo

Novartis·Phase 2·NCT03439839·16 enrolled

An Open Label, Multicenter Roll-over Extension Program (REP) to Characterize the Long-term Safety and Tolerability of Iptacopan (LNP023) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Completed PNH Phase 2 and Phase3 Studies With Iptacopan

Active, not recruiting· Novartis logo

Novartis·Phase 3·NCT04747613·208 enrolled

Age-Related Macular Degeneration1 trial

A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration

Active, not recruiting· Novartis logo

Novartis·Phase 2·NCT05230537·170 enrolled

Pediatric Patients1 trial

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

Recruiting· Novartis logo

Novartis·Phase 3·NCT06994845·31 enrolled

Thrombotic Microangiopathy1 trial

Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy

Not yet recruiting·FFirst Affiliated Hospital of Zhejiang University·N/A·NCT07347990·30 enrolled

13 more indications

Efficacy and Safety1 trial

Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

Completed· Novartis logo

Novartis·Phase 3·NCT04820530·40 enrolled

Hepatic Impairment1 trial

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.

Completed· Novartis logo

Novartis·Phase 1·NCT05078580·38 enrolled

Aged1 trial

Managed Access Programs for LNP023, Iptacopan

Available· Novartis logo

Novartis··NCT05222412

Efficacy1 trial

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

Terminated· Novartis logo

Novartis·Phase 2·NCT05086744·19 enrolled

Hemolytic Anemia1 trial

A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)

Not yet recruiting·BBing Han·N/A·NCT06847607·20 enrolled

Treatment1 trial

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

Completed· Novartis logo

Novartis·Phase 3·NCT05630001·52 enrolled

Kidney1 trial

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

Recruiting· Novartis logo

Novartis·Phase 2·NCT06797518·20 enrolled

C3G1 trial

A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Complement 3 Glomerulopathy.

Recruiting· Novartis logo

Novartis·Phase 3·NCT04817618·98 enrolled

IC-MPGN1 trial

A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Evaluate the Efficacy and Safety of Iptacopan (LNP023) in Idiopathic Immune-complex-mediated Membranoproliferative Glomerulonephritis (IC-MPGN)

Recruiting· Novartis logo

Novartis·Phase 3·NCT05755386·106 enrolled

Lupus Nephritis1 trial

An Adaptive, Randomized, Double-blind, Dose Exploration, Parallel Group, Placebo Controlled, Multicenter Phase 2 Trial to Evaluate the Efficacy, Safety and Tolerability of LNP023 in Combination With Standard-of-care With and Without Oral Corticosteroids in Patients With Active Lupus Nephritis Class III-IV, +/- V

Recruiting· Novartis logo

Novartis·Phase 2·NCT05268289·240 enrolled

Vasculitis1 trial

A Randomized, Controlled Study to Evaluate LNP023 (Iptacopan) in Patients With Active ANCA-associated Vasculitis

Active, not recruiting· Novartis logo

Novartis·Phase 2·NCT06388941·84 enrolled

Generalized Myasthenia Gravis1 trial

A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Iptacopan in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase

Recruiting· Novartis logo

Novartis·Phase 3·NCT06517758·146 enrolled

Hemoglobinuria, Paroxysmal1 trial

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

Active, not recruiting· Novartis logo

Novartis··NCT06903234·200 enrolled

Regulatory

FDA Approval Timeline[5]

Dec 5, 2023

FABHALTA

NDA NDA218276

Sources

References

[1]
ClinicalTrials.gov — 20 trial records for Iptacopan. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Fabhalta drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Iptacopan compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Iptacopan. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA218276). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Fabhalta. U.S. Food & Drug Administration.
[7]
PubMed — 10 publications indexed for Iptacopan. U.S. National Library of Medicine.
[8]
SEC EDGAR — Novartis company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Factor B Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: Novartis

Patent Landscape[6]

Patent Cliff

Jul 14, 2034

Earliest patent expiration — generic entry possible after this date

Patent #Expires

10093663Jul 14, 2034

9682968Jul 14, 2034

11723901Aug 30, 2038

12285422Aug 30, 2038

12384758May 17, 2041

3 more patents

11603363May 25, 2041

11951101Jul 15, 2041

12453726Jul 15, 2041

Key Publications[7]

Risk factors and temporal trends of early urological complications after kidney transplantation: a 15-year single-center cohort study.

Ren FailDec 1, 2026DOI: 10.1080/0886022X.2026.2678061

Pentoxifylline and cardiorenal outcomes in advanced CKD with untreated dyslipidemia: a longitudinal cohort study.

Ren FailDec 1, 2026DOI: 10.1080/0886022X.2026.2694246

Circulating Adipokines as Diagnostic and Prognostic Markers in Idiopathic Pulmonary Fibrosis.

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Correction: Endovascular therapy for erectile dysfunction: insights from more than 1000 treatments.

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Augmenting in vitro cancer cell toxicity via reactive oxygen species burst and glucose depletion induced by C14-Cu/Pt bimetallic nanozyme cascade reaction.

Mikrochim ActaJul 3, 2026DOI: 10.1007/s00604-026-08228-8

Phototoxicity of brightfield live-cell imaging on murine ovarian follicles.

Arch Gynecol ObstetJul 3, 2026DOI: 10.1007/s00404-026-08515-y

Role of NADPH oxidase generated O(2)(-) in seed germination and cold stress response of Brassica napus.

ProtoplasmaJul 3, 2026DOI: 10.1007/s00709-026-02240-8

Cost-effectiveness of community versus hospital-based mental healthcare for severe mental illness in South-East Europe: economic evaluation of five randomised trials.

Eur J Health EconJul 3, 2026DOI: 10.1007/s10198-026-01960-1

Antibiotic stewardship in non-perforated gangrenous appendicitis: outcomes of single-dose versus postoperative therapy in a retrospective cohort study.

Langenbecks Arch SurgJul 3, 2026DOI: 10.1007/s00423-026-04129-9

Interventions to improve the psychosocial outcomes of individuals with lymphedema: a systematic review.

Support Care CancerJul 3, 2026DOI: 10.1007/s00520-026-10972-9

Fabhalta

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q3 2026

Q4 2026

Q1 2027

Q4 2027

Trials by Indication[1]

Atypical Hemolytic-Uremic Syndrome2 trials

Paroxysmal Nocturnal Hemoglobinuria2 trials

Age-Related Macular Degeneration1 trial

Pediatric Patients1 trial

Thrombotic Microangiopathy1 trial

Efficacy and Safety1 trial

Hepatic Impairment1 trial

Aged1 trial

Efficacy1 trial

Hemolytic Anemia1 trial

Treatment1 trial

Kidney1 trial

C3G1 trial

IC-MPGN1 trial

Lupus Nephritis1 trial

Vasculitis1 trial

Generalized Myasthenia Gravis1 trial

Hemoglobinuria, Paroxysmal1 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

Patent Landscape[6]

Key Publications[7]

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