Iptacopan
Also known as Fabhalta, Iptacopan hydrochloride
NOVARTIS AG NVS NVSEF · Atypical Hemolytic Uremic Syndrome; Paroxysmal Nocturnal Hemoglobinuria; C3G; Generalized Myasthenia Gravis; IC-MPGN
Key figures
Next catalyst
None scheduled
Only dates with a source are listed
Phase
Approved
Not recorded
Linked trials
9
From ClinicalTrials.gov
Latest FDA action
Approved
NDA218276,
Recalls, shortages, patents and EU status at a glance
Key information
- Sponsor
- NOVARTIS AG NVS NVSEF Source: Company record for NOVARTIS AG (opens in a new tab)
- Indications
- Atypical Hemolytic Uremic Syndrome; Paroxysmal Nocturnal Hemoglobinuria; C3G; Generalized Myasthenia Gravis; IC-MPGN Source: Conditions from ClinicalTrials.gov (opens in a new tab)
- Application
- NDA 218276 · Priority review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for NDA 218276 (opens in a new tab)
- Designations
- None recorded
- Next catalyst
- None scheduled
Development timeline
- Preclinical Completed
- Phase 1 Completed
- Phase 2 Completed
- Phase 3 CompletedFirst trial start 19 Jul 20219 linked trials
- Filed Completed
- Approved Current stageFirst approval 5 Dec 2023
Overview
- Mechanism
- Not recorded
- Route
- Not recorded
- Modality
- Not recorded
- Specialties
- Not recorded
Overview text is written by HealthTrade editors.
Key dates
No catalysts yet. Editors add each date together with its source.
FDA designations
No FDA designations are on record for Iptacopan. Editors add each one with its source.
Filed application
No filed application is on record for Iptacopan. Approvals are listed under FDA actions.
FDA advisory committees
No FDA advisory committee meetings are on record for Iptacopan.
Clinical trials
| Trial | Title | Phase | Status | Enrollment | Primary completion | Results posted |
|---|---|---|---|---|---|---|
| NCT06994845ClinicalTrials.gov record for NCT06994845 (opens in a new tab) | Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN | Phase 3 | Recruiting | 31 (estimated) | 28 Jun 2030 (estimated) | No |
| NCT06517758ClinicalTrials.gov record for NCT06517758 (opens in a new tab) | A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG. | Phase 3 | Active, not recruiting | 146 (estimated) | 30 Apr 2027 (estimated) | No |
| NCT05935215ClinicalTrials.gov record for NCT05935215 (opens in a new tab) | Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS) | Phase 3 | Recruiting | 50 (estimated) | 21 Jul 2028 (estimated) | No |
| NCT05755386ClinicalTrials.gov record for NCT05755386 (opens in a new tab)APPARENT | Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN | Phase 3 | Recruiting | 106 (estimated) | 27 Oct 2028 (estimated) | No |
| NCT05630001ClinicalTrials.gov record for NCT05630001 (opens in a new tab)APPULSE | Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. | Phase 3 | Completed | 52 (actual) | 17 Oct 2024 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT05630001 (opens in a new tab) |
Posted results on ClinicalTrials.gov for APPULSE
Serious adverse events (Adverse events were reported from first dose of study treatment until end of study treatment plus 7 days post treatment for non-serious AEs and 30 days post treatment for serious AEs if patient did not roll over to REP study, up to a maximum duration of 198 days): LNP023 200mg b.i.d. 2 of 52 Source: ClinicalTrials.gov results for NCT05630001 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. | ||||||
| NCT04889430ClinicalTrials.gov record for NCT04889430 (opens in a new tab)APPELHUS | Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy | Phase 3 | Completed | 34 (actual) | 11 Apr 2026 (actual) | No |
| NCT04817618ClinicalTrials.gov record for NCT04817618 (opens in a new tab)APPEAR-C3G | Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. | Phase 3 | Recruiting | 98 (estimated) | 29 Jan 2027 (estimated) | No |
Publications listing APPEAR-C3G (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT04747613ClinicalTrials.gov record for NCT04747613 (opens in a new tab) | Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Active, not recruiting | 208 (actual) | 18 Oct 2027 (estimated) | No |
Publications listing NCT04747613 (1), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
| NCT04820530ClinicalTrials.gov record for NCT04820530 (opens in a new tab)APPOINT-PNH | Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy | Phase 3 | Completed | 40 (actual) | 2 Nov 2022 (actual) | Yes First posted Results on ClinicalTrials.gov for NCT04820530 (opens in a new tab) |
Posted results on ClinicalTrials.gov for APPOINT-PNH Serious adverse events (Adverse events of LNP023 group were reported from first dose of study treatment until the end of study treatment plus up to 30 days, up to a maximum duration of 48 weeks): LNP023 200mg b.i.d. 8 of 40 Source: ClinicalTrials.gov results for NCT04820530 (opens in a new tab)· imported Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately. Publications listing APPOINT-PNH (3), newest first
Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies. | ||||||
Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.
Trial quality
About CT-VCALoading trial quality…
FDA actions
| Application | Action | Date | Outcome | Review | Source |
|---|---|---|---|---|---|
| NDA218276Action letter for NDA218276 (opens in a new tab) | Supplement 7 Efficacy | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA218276Action letter for NDA218276 (opens in a new tab) | Supplement 4 Efficacy | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA218276Action letter for NDA218276 (opens in a new tab) | Supplement 1 Efficacy | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) | |
| NDA218276Action letter for NDA218276 (opens in a new tab) | Original 1 Type 1 - New Molecular Entity | Approved | priority | Drugs@FDA via openFDA (opens in a new tab) |
Data provided by the U.S. Food and Drug Administration.
FDA recalls load here.
FDA drug shortages load here.
Orange Book patents load here.
FAERS adverse event counts load here.
Papers and preprints load here.
Community discussion loads here.
EU status (EMA)
AuthorisedOrphan medicinePRIME
Fabhalta (EMEA/H/C/005764)
- Authorised
- Therapeutic area
- Hemoglobinuria, Paroxysmal
- Marketing authorisation holder
- Novartis Europharm Limited
- Active substance (INN)
- iptacopan
- ATC code
- L04A
- Latest EMA decision
Matched by active substance: EMA's INN or active substance equals this drug's name.
EMA page for Fabhalta (opens in a new tab)EMA medicine data (opens in a new tab)
Source: European Medicines Agency (EMA), medicines data download. Licence: EMA re-use with acknowledgement. EMA medicines report 2026-10-26.
Related news
No recent headlines mention this drug.
Sources
- Drugs@FDA 218276 (opens in a new tab) Licence: public-domain. Official registry record this listing came from
- ClinicalTrials.gov NCT06994845 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT06517758 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05935215 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05755386 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT05630001 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04889430 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04817618 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04747613 (opens in a new tab) Licence: public-domain. Fetched
- ClinicalTrials.gov NCT04820530 (opens in a new tab) Licence: public-domain. Fetched
- Drugs@FDA via openFDA NDA218276 (opens in a new tab) Licence: cc0. Fetched
Record updated .
Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.