Iptacopan

Overview

Iptacopan is under investigation in clinical trial NCT04578834 (Study of Efficacy and Safety of LNP023 in Primary Iga Nephropathy Patients).

Indication

用于治疗既往未接受过补体抑制剂（如依库珠单抗等）治疗的阵发性睡眠性血红蛋白尿症(PNH，一种罕见病)成人患者。 PNH是一种由补体介导的罕见血液疾病。PNH患者的造血干细胞PIG-A基因发生突变，导致其产生易于被补体系统过早破坏的红细胞，从而引发血管内溶血（红细胞在血管内被破坏）和血管外溶血（红细胞在脾脏和肝脏中被破坏），临床主要表现为贫血、阵发性血红蛋白尿、骨髓造血功能衰竭和血栓形成等。

Associated Conditions

No associated conditions information available.

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN	2025/05/29	NCT06994845	Phase 3	Not yet recruiting	Novartis Pharmaceuticals
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients	2025/04/18	NCT06934967	Phase 3	Not yet recruiting	Novartis Pharmaceuticals
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China	2025/04/17	NCT06931691	N/A	Recruiting	Novartis Pharmaceuticals
Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry	2025/03/30	NCT06903234	N/A	Active, not recruiting	Novartis Pharmaceuticals
A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)	2025/02/26	NCT06847607	Not Applicable	Not yet recruiting	Bing Han
Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN	2025/01/28	NCT06797518	Phase 2	Recruiting	Novartis Pharmaceuticals
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG.	2024/07/24	NCT06517758	Phase 3	Recruiting	Novartis Pharmaceuticals
Iptacopan in Patients With ANCA Associated Vasculitis	2024/04/29	NCT06388941	Phase 2	Recruiting	Novartis Pharmaceuticals
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)	2023/07/07	NCT05935215	Phase 3	Recruiting	Novartis Pharmaceuticals
Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN	2023/03/06	NCT05755386	Phase 3	Recruiting	Novartis Pharmaceuticals

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
FABHALTA	Novartis Pharmaceuticals Corporation	0078-1189	ORAL	200 mg in 1 1	12/5/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Fabhalta	Novartis Europharm Limited,Vista Building,Elm Park,Merrion Road,Dublin 4,D04 A9N6,Ireland	Authorised	5/17/2024

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
Iptacopan Hydrochloride Capsules	Novartis Pharma Schweiz AG	国药准字HJ20240016	化学药品	胶囊剂	4/24/2024

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
♐ FABHALTA CAPSULES 200MG	N/A	NOVARTIS PHARMACEUTICALS (HK) LIMITED	N/A	N/A	10/10/2024

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
FABHALTA iptacopan 200 mg hard capsules blister pack	410830	Novartis Pharmaceuticals Australia Pty Ltd	Medicine	A	8/12/2024

Related News

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NovelMed's Ruxoprubart demonstrated significant efficacy in a Phase II trial for treatment-naïve PNH patients, meeting all primary endpoints including complete transfusion avoidance and increased hemoglobin levels.

CDSCO Approves Novartis' Protocol Amendment for Iptacopan Study in Rare Kidney Disorders

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India's CDSCO expert committee has approved Novartis Healthcare's protocol amendment for a Phase IIIb extension study of iptacopan in patients with C3 glomerulopathy and idiopathic immune-complex-membranoproliferative glomerulonephritis.

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FABHALTA (iptacopan), Novartis' first-in-class oral factor B inhibitor, has received FDA approvals for paroxysmal nocturnal hemoglobinuria (PNH), IgA nephropathy (IgAN), and C3 glomerulopathy (C3G), marking a significant shift from traditional intravenous therapies.

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