MedPath

Iptacopan

Generic Name
Iptacopan
Brand Names
Fabhalta
Drug Type
Small Molecule
Chemical Formula
C25H30N2O4
CAS Number
1644670-37-0
Unique Ingredient Identifier
8E05T07Z6W
Background

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
-
Associated Therapies
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biospace.com
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New Phase IIIB data shows Novartis Fabhalta® improved hemoglobin levels in adult

In the Phase IIIB APPULSE-PNH study, oral Fabhalta® (iptacopan) improved average hemoglobin levels in adult PNH patients switched from anti-C5 therapies. PNH is a rare, chronic blood disorder often treated with anti-C5 therapies, and Fabhalta has shown efficacy and safety in previous Phase III trials. Novartis announced positive results from APPULSE-PNH, with Fabhalta's safety profile consistent with prior data.
globenewswire.com
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New Phase IIIB data shows Novartis Fabhalta® improved

In the Phase IIIB APPULSE-PNH study, oral Fabhalta® (iptacopan) improved average hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH) switched from anti-C5 therapies.
biospace.com
·

NovelMed Receives FDA IND Approval for NM8074 (Ruxoprubart), the First Anti-Bb

FDA approves Phase II trial for Ruxoprubart, a selective alternative pathway inhibitor, in IgAN patients, with potential to treat multiple disorders. Ruxoprubart's unique mechanism avoids blocking the classical pathway, offering advantages over existing treatments.
globenewswire.com
·

NovelMed Receives FDA IND Approval for NM8074

FDA clears Ruxoprubart for Phase II IgAN trial, a selective AP inhibitor with potential in multiple diseases, including PNH, C3G, aHUS, AAV, and IgAN, offering a safer alternative to existing complement blockers.
pharmabiz.com
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Novartis highlights new 96-week results from phase III Scemblix ASC4FIRST trial at ASH

Novartis presents over 65 abstracts at ASH and SABCS, highlighting new data on Scemblix, Kisqali, and other treatments, emphasizing commitment to cancer and blood disorder patients. The company also showcases personal breast cancer stories at SABCS, aiming to uplift and unite the community.
biospace.com
·

Novartis highlights new 96-week results from Phase III Scemblix® ASC4FIRST trial at ASH

Novartis to present over 65 abstracts at ASH and SABCS, including 96-week Scemblix results and Kisqali 4-year analysis. Data highlights commitment to cancer and blood disorder treatments.
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