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 20 indications[4].[1]

Fabhalta is one of 169 small molecule programs targeting IgA Nephropathy. It is currently in Approved.

Indications

Total Trials

Active Trials

168

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Iga NephropathyApproved

Atypical Hemolytic-Uremic SyndromeRecruiting

Hemolytic AnemiaActive, not recruiting

Abnormality of the KidneyActive, not recruiting

IGA GlomerulonephritisApproved

Liver DiseaseApproved

GlomerulonephritisApproved

Primary Membranoproliferative GlomerulonephritisApproved

Complement 3 GlomerulopathyApproved

Dense Deposit DiseaseApproved

Iga NephropathyApproved

Approved

Atypical Hemolytic-Uremic SyndromeApproved

Recruiting

Hemolytic AnemiaPreclinical

Active, not recruiting

Abnormality of the KidneyPreclinical

Active, not recruiting

IGA GlomerulonephritisApproved

Approved

Liver DiseaseApproved

Approved

GlomerulonephritisApproved

Approved

Primary Membranoproliferative GlomerulonephritisApproved

Approved

Complement 3 GlomerulopathyApproved

Approved

Dense Deposit DiseaseApproved

Approved

Showing 10 of 20 indications

Competition

Competitive Landscape

100 competitors in Liver Disease

#DrugCompanyPhaseTrials

Fabhalta

Novartis

Approved

20 2

Elunate

Takeda

Approved

—3

Incivek

Vertex Pharmaceuticals

Tukysa

Veozah

Sovaldi

Kalydeco

Vertex Pharmaceuticals

Piqray

Balversa

Cibinqo

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

Timeline

Expected Milestones

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

Glomerulonephritis, IGA

NCT06797518

Q3 2028

Phase 2Recruiting

Lupus Nephritis

NCT05268289

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

A Multicenter, Single-arm, Open Label Trial to Evaluate Efficacy and Safety of Oral, Twice Daily LNP023 in Adult aHUS Patients Who Are Naive to Complement Inhibitor Therapy

Completed· 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

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

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

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

13 more indications

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

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

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

Primary Immunoglobulin A Nephropathy1 trial

A Single-arm, Multicenter, Phase III Study to Assess Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients of 2 to <18 Years of Age With Primary Immunoglobulin A Nephropathy (IgAN)

Recruiting· Novartis logo

Novartis·Phase 3·NCT06994845·31 enrolled

Thrombotic Microangiopathy1 trial

A Prospective, Multicenter, Single-Arm Study: Safety and Efficacy of Iptacopan in the Treatment of High-Risk Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (TA-TMA)

Recruiting·FFirst Affiliated Hospital of Zhejiang University·Preclinical·NCT07347990·30 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

Active, not recruiting· Novartis logo

Novartis·Phase 3·NCT06517758·146 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

Glomerulonephritis, IGA1 trial

A Multicenter, Single Arm, Open Label Biopsy Study to Evaluate Structural and Functional Changes in Kidneys of Adult Patients With IgA Nephropathy Receiving Iptacopan on Top of Supportive Care

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

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

Dec 5, 2023

FABHALTA

NDA NDA218276

Mar 26, 2024

FABHALTA

NDA NDA218276

May 24, 2024

FABHALTA

NDA NDA218276

Mar 20, 2025

FABHALTA

NDA NDA218276

Jul 16, 2026

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]

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

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

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

Intracerebral Glutamate and Glycerol in Severe Acute Brain Injury: Retrospective Study of Association With Neuroworsening and Outcome.

Acta Anaesthesiol ScandOct 1, 2026DOI: 10.1111/aas.70333

Local Anaesthetic Dosing and Toxicity Knowledge in Australian Emergency Departments: A Multicentre Cross-Sectional Survey.

Emerg Med AustralasOct 1, 2026DOI: 10.1111/1742-6723.70352

Corrigendum to "MIF-CD74 axis facilitates MDSC infiltration in the tumor microenvironment of pancreatic ductal adenocarcinoma" [Cancer Lett. 645, (2026), 218348].

Cancer LettSep 10, 2026DOI: 10.1016/j.canlet.2026.218794

Early orthopedic treatment of skeletal Class III malocclusion: Correlating dentoskeletal changes with psychosocial outcomes using the modified SEC III protocol and PIDAQ.

J World Fed OrthodSep 10, 2026DOI: 10.1016/j.ejwf.2026.07.004

Sequential disease activity assessment for predicting future treatment trajectories in patients with rheumatoid arthritis treated with JAK inhibitors: a group-based trajectory and landmark analysis.

Ann Rheum DisSep 10, 2026DOI: 10.1016/j.ard.2026.08.010

Re: Light A, Peters M, Gopalakrishnan A, et al. Oncological Outcomes Following Focal HIFU and Cryotherapy for Treatment of Nonmetastatic Prostate Cancer in the United Kingdom: An Updated Analysis of 3477 Patients from the Prospective HEAT and ICE Registries. Eur Urol. In press. http://dx.doi.org/10.1016/j.eururo.2026.05.007.

Eur UrolSep 10, 2026DOI: 10.1016/j.eururo.2026.07.038

Early Hemodynamic Trajectory Phenotypes and Exploratory Albumin Exposure-Mortality Associations in Septic Shock: A Retrospective Cohort Study.

Clin TherSep 10, 2026DOI: 10.1016/j.clinthera.2026.08.011

Effects of Phosphodiesterase-5 Inhibitors on Mortality and Clinical Outcomes in Advanced Interstitial Lung Disease with Severe Impairment in Gas Exchange: A Systematic Review and Meta-Analysis.

Clin TherSep 10, 2026DOI: 10.1016/j.clinthera.2026.08.010

Fabhalta

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2027

Q4 2027

Q3 2028

Trials by Indication[1]

Atypical Hemolytic-Uremic Syndrome2 trials

Paroxysmal Nocturnal Hemoglobinuria2 trials

Hemolytic Anemia1 trial

Hepatic Impairment1 trial

Efficacy and Safety1 trial

Age-Related Macular Degeneration1 trial

Aged1 trial

Efficacy1 trial

Treatment1 trial

Primary Immunoglobulin A Nephropathy1 trial

Thrombotic Microangiopathy1 trial

Vasculitis1 trial

Generalized Myasthenia Gravis1 trial

Lupus Nephritis1 trial

Glomerulonephritis, IGA1 trial

C3G1 trial

IC-MPGN1 trial

Hemoglobinuria, Paroxysmal1 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

Patent Landscape[6]

Key Publications[7]

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