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
- -
FDA Approves Expanded Use Of Novartis' Rare Disease Drug Fabhalta For Kidney Disease
FDA approved Novartis' Fabhalta for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid progression. Approval based on Phase 3 APPLAUSE-IgAN study showing a 44% proteinuria reduction at nine months. Continued approval may depend on further study results expected in 2025. IgAN is a rare kidney disease affecting about 25 per million annually. Novartis is also developing two other IgAN therapies.
Pharma Stock Roundup: ABBV to Buy CERE, JNJ's 2024 View, LLY & NVS Get FDA Nod
AbbVie to acquire Cerevel Therapeutics for $8.7B, enhancing its neuroscience pipeline. FDA approves Novartis' Fabhalta for PNH and Lilly's Jaypirca for second leukemia indication. Pfizer halts phase III for danuglipron due to side effects. J&J forecasts sales growth, Roche to buy Carmot Therapeutics for obesity treatments.
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