Novartis’ Fabhalta Receives FDA Approval for IgA Nephropathy, Expanding Its Potential Blockbuster Indications

Aug 11, 2024 07:41 CST Updated 07:41

Novartis

Drug Development and Manufacturing

▎WuXiEdited by Kant Content Team

News on August 8,FDA Approves Novartis' Potential Blockbuster Therapy Fabhalta (iptacopan) for Expanded Indication to Reduce Proteinuria Levels in Adult Patients with IgA Nephropathy (IgAN)These patients are at risk of rapid disease progression. A previous press release from Novartis stated,Fabhalta isThe First IgAN Therapy Targeting the Alternative Complement Pathway。

It is worth mentioning that, just the day before (August 7),Announcement on the official website of China NMPA,iptacopan（New Indication Marketing Application for Iptacopan Hydrochloride CapsulesAccepted for Filing. This is the second indication marketing application for this product in China.

This FDA approval is mainly based on the positive results of the randomized, double-blind, placebo-controlled Phase 3 clinical trial APPLAUSE-IgAN. This trial evaluated the efficacy and safety of Fabhalta in 518 adult patients with primary IgAN. The two primary endpoints of the study were the patients' 24-hour urine protein-to-creatinine ratio (UPCR) and the estimated glomerular filtration rate (eGFR) slope over 24 months.

Previously released interim analysis data shows,Compared with patients receiving placebo and supportive care, patients in the Fabhalta treatment group had a 38.3% reduction in proteinuria after 9 months of treatment (p<0.0001), reaching the primary endpoint of the trial.Another primary endpoint of the trial is to evaluate Fabhalta's ability to slow the progression of IgAN by measuring the annual eGFR slope over 24 months, with the results expected to be announced upon completion of the study in 2025. The study also demonstrated that Fabhalta has a favorable safety and tolerability profile, consistent with previously reported safety characteristics.

Fabhalta is an oral specific alternative complement pathway factor B inhibitor., which can efficiently inhibit factor B in the alternative complement pathway. It may treat various diseases caused by abnormal alternative pathway function without affecting the immune response to microbial invasion mediated by other complement pathways, reducing the risk of infection for patients.

▲Introduction to Fabhalta (iptacopan) (Source of image:Novartis official website)

Fabhalta is one of Novartis' key development projects, with the potential to treat a variety of indications.It was approved by the United States in December 2023.FDA ApprovalLaunches as the First Oral Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Adults.

The interim results of the previously announced Phase 3 clinical trial APPEAR-C3G showed,In patients with C3 glomerulopathy (C3G), Fabhalta reduced proteinuria levels by 35% at 6 months of treatment compared to placebo.Novartis stated in its Q2 2024 financial report that the efficacy of Fabhalta at 12 months of treatment was consistent with that at 6 months, with specific data to be released at an upcoming medical conference.Novartis is expected to submit a regulatory application for Fabhalta in the treatment of C3G in the second half of 2024.

▲Fabhalta significantly reduces proteinuria levels in patients with C3G (Image source:Novartis official website)

Novartis is also conducting multiple clinical trials to evaluate the efficacy of Fabhalta in treating lupus nephritis, immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN), atypical hemolytic uremic syndrome (aHUS), myasthenia gravis, and other indications.This therapy was once listed by the industry media Evaluate as10 Potential Blockbuster Therapies Worth WatchingOne of.

References:

[1] Fabhalta label. Retrieved August 7, 2024, from https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/218276s001lbl.pdf

[2] Q2 2024 Results. Retrieved August 7, 2024, from https://www.novartis.com/sites/novartis_com/files/q2-2024-investor-presentation.pdf

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