Iptacopan History and Updates

69 events · 2018 – present

Synced daily from openFDA and ClinicalTrials.gov. Last sync: August 17, 2026.

FDA Approvals

Label Revisions

Trial Milestones

Pivotal Publications

Source: openFDA, ClinicalTrials.gov via AACT, PubMed · Hipa.ai, 2026-08-13Download chart as PNG

This page tracks every FDA action, clinical trial milestone, and major publication for Iptacopan since 2018, drawn from openFDA, ClinicalTrials.gov, and PubMed.

All published updates (6)

2026

Label revisionFABHALTA Boxed Warning Revised for Serious Encapsulated Bacterial Infections· Jul 16, 2026
Label revisionFabhalta (Iptacopan) Label Receives Boxed Warning for Serious Infections· Jun 8, 2026
Primary completionIptacopan Phase 3 Trial for Atypical Hemolytic Uremic Syndrome Completes· Apr 11, 2026

2025

Results postedIptacopan Phase 3 Trial Shows Improved Hemoglobin in PNH Patients· Dec 15, 2025
FDA approval (supplemental)Fabhalta (Iptacopan) Receives FDA Supplemental Approval for Efficacy· Mar 20, 2025
Results postedIptacopan Phase 2 Trial Shows Response in Cold Agglutinin Disease· Mar 19, 2025

Upcoming & expected milestones

Primary completionOct 1, 2026· ClinicalTrials.gov

Phase 2 primary completion: NCT05230537

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

Primary completionDec 30, 2026· ClinicalTrials.gov

Phase 2 primary completion: NCT06388941

Iptacopan in Patients With ANCA Associated Vasculitis

Primary completionJan 29, 2027· ClinicalTrials.gov

Phase 3 primary completion: NCT04817618

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

Primary completionApr 30, 2027· ClinicalTrials.gov

Phase 3 primary completion: NCT06517758

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

Primary completionOct 18, 2027· ClinicalTrials.gov

Phase 3 primary completion: NCT04747613

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

Primary completionNov 27, 2027· ClinicalTrials.gov

Phase 2 primary completion: NCT06797518

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

Primary completionDec 31, 2027· ClinicalTrials.gov

NA primary completion: NCT07347990

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

Primary completionJul 21, 2028· ClinicalTrials.gov

Phase 3 primary completion: NCT05935215

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

Primary completionOct 27, 2028· ClinicalTrials.gov

Phase 3 primary completion: NCT05755386

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

Primary completionOct 1, 2029· ClinicalTrials.gov

Primary completion: NCT06903234

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

Primary completionJun 28, 2030· ClinicalTrials.gov

Phase 3 primary completion: NCT06994845

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

Past events

2026

Label revisionJul 16, 2026· FDA

Boxed warning revised on FABHALTA label (effective 2026-07-16)

WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA FABHALTA, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae, Neisseria meningitid

Label revisionJun 8, 2026· FDA

Boxed warning present on label on FABHALTA label (effective 2026-06-08)

Primary completionApr 11, 2026· ClinicalTrials.gov

Phase 3 primary completion: NCT04889430

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

Primary completionMar 19, 2026· ClinicalTrials.gov

Phase 2 primary completion: NCT05268289

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

Trial registeredJan 16, 2026· ClinicalTrials.gov

NA trial registered on ClinicalTrials.gov: NCT07347990

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

Trial startedJan 1, 2026· ClinicalTrials.gov

NA trial started: NCT07347990

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

2025

Results postedDec 15, 2025· ClinicalTrials.gov

Trial results posted: NCT05630001

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

Trial startedNov 27, 2025· ClinicalTrials.gov

Phase 3 trial started: NCT06994845

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

Primary completionOct 30, 2025· ClinicalTrials.gov

NA primary completion: NCT06847607

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

Trial registeredMay 29, 2025· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT06994845

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

Trial startedMar 31, 2025· ClinicalTrials.gov

Trial started: NCT06903234

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

Trial registeredMar 30, 2025· ClinicalTrials.gov

Trial registered on ClinicalTrials.gov: NCT06903234

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

FDA approval (supplemental)Mar 20, 2025· FDA

FDA approved supplemental application for Fabhalta (iptacopan) — Efficacy (NDA 218276 S-4)

Sponsor: NOVARTIS. Class: Efficacy.

Results postedMar 19, 2025· ClinicalTrials.gov

Trial results posted: NCT05086744

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

Trial startedMar 1, 2025· ClinicalTrials.gov

NA trial started: NCT06847607

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

Trial registeredFeb 26, 2025· ClinicalTrials.gov

NA trial registered on ClinicalTrials.gov: NCT06847607

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

Trial startedFeb 21, 2025· ClinicalTrials.gov

Phase 2 trial started: NCT06797518

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

Trial registeredJan 28, 2025· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT06797518

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

2024

Primary completionOct 17, 2024· ClinicalTrials.gov

Phase 3 primary completion: NCT05630001

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

FDA approval (supplemental)Aug 7, 2024· FDA

FDA approved supplemental application for Fabhalta (iptacopan) — Efficacy (NDA 218276 S-1)

Sponsor: NOVARTIS. Class: Efficacy.

Trial startedAug 5, 2024· ClinicalTrials.gov

Phase 2 trial started: NCT06388941

Iptacopan in Patients With ANCA Associated Vasculitis

Trial startedJul 31, 2024· ClinicalTrials.gov

Phase 3 trial started: NCT06517758

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

Trial registeredJul 24, 2024· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT06517758

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

FDA approval (supplemental)May 24, 2024· FDA

FDA approved supplemental application for Fabhalta (iptacopan) — REMS (NDA 218276 S-3)

Sponsor: NOVARTIS. Class: REMS.

Trial registeredApr 29, 2024· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT06388941

Iptacopan in Patients With ANCA Associated Vasculitis

FDA approval (supplemental)Mar 26, 2024· FDA

FDA approved supplemental application for Fabhalta (iptacopan) — Labeling (NDA 218276 S-2)

Sponsor: NOVARTIS. Class: Labeling.

Trial startedFeb 28, 2024· ClinicalTrials.gov

Phase 3 trial started: NCT05935215

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

Results postedJan 3, 2024· ClinicalTrials.gov

Trial results posted: NCT03439839

Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobin

2023

FDA approvalDec 5, 2023· FDA

FDA approved Fabhalta (iptacopan) (NDA 218276)

Sponsor: NOVARTIS. Class: Type 1 - New Molecular Entity.

Results postedNov 15, 2023· ClinicalTrials.gov

Trial results posted: NCT04820530

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

Trial startedOct 2, 2023· ClinicalTrials.gov

Phase 3 trial started: NCT05755386

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

Primary completionSep 20, 2023· ClinicalTrials.gov

Phase 2 primary completion: NCT05086744

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

Trial registeredJul 7, 2023· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT05935215

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

Trial startedApr 24, 2023· ClinicalTrials.gov

Phase 3 trial started: NCT05630001

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

Trial registeredMar 6, 2023· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT05755386

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

2022

Trial registeredNov 29, 2022· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT05630001

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

Primary completionNov 2, 2022· ClinicalTrials.gov

Phase 3 primary completion: NCT04820530

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

Trial startedAug 10, 2022· ClinicalTrials.gov

Phase 2 trial started: NCT05268289

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

Primary completionJun 20, 2022· ClinicalTrials.gov

Phase 1 primary completion: NCT05078580

Pharmacokinetic (PK) and Safety Study of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to M

Trial registeredMar 7, 2022· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT05268289

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

Trial startedFeb 17, 2022· ClinicalTrials.gov

Phase 2 trial started: NCT05230537

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

Trial registeredFeb 9, 2022· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT05230537

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

Trial registeredFeb 3, 2022· ClinicalTrials.gov

Trial registered on ClinicalTrials.gov: NCT05222412

Managed Access Programs for LNP023, Iptacopan

Trial startedJan 17, 2022· ClinicalTrials.gov

Phase 3 trial started: NCT04889430

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

2021

Trial startedDec 21, 2021· ClinicalTrials.gov

Phase 2 trial started: NCT05086744

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

Trial startedNov 10, 2021· ClinicalTrials.gov

Phase 1 trial started: NCT05078580

Pharmacokinetic (PK) and Safety Study of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to M

Trial registeredOct 21, 2021· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT05086744

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

Trial registeredOct 14, 2021· ClinicalTrials.gov

Phase 1 trial registered on ClinicalTrials.gov: NCT05078580

Pharmacokinetic (PK) and Safety Study of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to M

Trial startedJul 28, 2021· ClinicalTrials.gov

Phase 3 trial started: NCT04817618

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

Trial startedJul 27, 2021· ClinicalTrials.gov

Phase 3 trial started: NCT04747613

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

Trial startedJul 19, 2021· ClinicalTrials.gov

Phase 3 trial started: NCT04820530

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

Trial registeredMay 17, 2021· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT04889430

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

Trial registeredMar 29, 2021· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT04820530

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

Trial registeredMar 26, 2021· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT04817618

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

Trial registeredFeb 10, 2021· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT04747613

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

2020

Primary completionApr 22, 2020· ClinicalTrials.gov

Phase 2 primary completion: NCT03439839

Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobin

2018

Trial startedApr 9, 2018· ClinicalTrials.gov

Phase 2 trial started: NCT03439839

Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobin

Trial registeredFeb 20, 2018· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT03439839

Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobin

Sources

FDA approvals, label revisions, and recalls come from the public openFDA APIs (Drugs@FDA, Drug Label, Drug Enforcement). Drug-safety communications come from the FDA RSS feed. Trial milestones come from ClinicalTrials.gov via the AACT pg dump. Pivotal-publication events come from PubMed, filtered to high-impact journals linked to a Phase 3 trial in our inventory.