Iptacopan hydrochloride is under clinical development by Novartis and currently in Phase II for Acquired (Autoimmune) Hemolytic Anemia. According to GlobalData, Phase II drugs for Acquired (Autoimmune) Hemolytic Anemia have a 62% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Iptacopan hydrochloride’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Iptacopan hydrochloride overview

Iptacopan hydrochloride (Fabhalta) is a factor B inhibitor of the alternative complement pathway15-17. It is formulated as hard gelatin capsules for oral route of administration. It is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).

Iptacopan (LNP-023) is under development for the treatment of glomerulonephritis- IgA nephropathy (Berger's disease), early to middle age-related macular degeneration, C3 glomerulopathy, proteinuria, atypical hemolytic uraemic syndrome, lupus nephritis, immune complex membranoproliferative glomerulonephritis, paroxysmal nocturnal hemoglobinuria and anti-neutrophil cytoplasmic antibody-associated vasculitis (ANCA Vasculitis). It is administered through oral route as a hard gelatin capsule. It acts by targeting complement factor B (FB). The therapeutic candidate is a new molecular entity (NME).

It was also under development for the treatment of idiopathic/primary membranous nephropathy.

Novartis overview

Novartis is a healthcare company that focuses on the discovery, development, manufacture and marketing of prescription and generic pharmaceutical products and eye care products. It provides drugs for the treatment of cancer, cardiovascular diseases, dermatological conditions, neurological disorders, ophthalmic and respiratory diseases, hematologic diseases, solid tumors, immune disorders, and infections, among others. Novartis conducts research in various disease areas through the BioMedical Research division. The company operates through a network of subsidiaries and offices across the Americas, Europe, the Middle East, Africa and Asia-Pacific. Novartis is headquartered in Basel, Switzerland.

For a complete picture of Iptacopan hydrochloride’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 10 June 2024

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GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.