Iptacopan Hydrochloride

iStock, mohd izzuan Novartis will continue to study Fabhalta in other indications, with late-stage studies in two rare diseases—atypical haemolytic uraemic syndrome and generalized myasthenia gravis—expected to read out later this year and next year, respectively. Novartis has pulled the plug on a Phase 2 study testing the complement blocker Fabhalta for patients with lupus that has inflamed the kidneys, a condition called lupus nephritis. “The decision was based on strategic considerations and was not driven by any interim efficacy or safety findings,” a company spokesperson told BioSpace in an email. The study enrolled 81 patients with lupus nephritis, according to a federal clinical trials database . The mid-stage study was originally designed to have two parts, this first of which compared Fabhalta, also known as iptacopan, plus standard of care against placebo plus standard of care. The second part was expected to have three arms testing iptacopan plus standard of care, iptacopan plus placebo and placebo plus standard of care. The primary outcome for both parts was percentage of patients achieving a complete renal response at week 24. Novartis has completed the first portion of the study but has decided not to continue on with part two, the spokesperson said. Fabhalta was first approved in late 2023 for a rare blood disorder called paroxysmal nocturnal hemoglobinuria. The drug is a complement factor B inhibitor that tunes down activity of an immune pathway thought to contribute to disease activity when overactivated. In the following years, Fabhalta has also secured approvals in IgA nephropathy and complement 3 glomerulopathy . Last year, Novartis reported $505 million in global sales for Fabhalta, up nearly 290% year-on-year, with growth driven by the drug’s continued strong uptake across indications. Despite the termination of the mid-stage lupus nephritis study, “[i]ptacopan studies in other indications will continue as planned,” the spokesperson told BioSpace . Novartis has two Phase 3 studies for the drug in atypical haemolytic uraemic syndrome, with one readout planned for this year and another for 2028, according to the pharma’s Q2 report . Fabhalta is also being studied for generalized myasthenia gravis, for which a late-stage data drop is expected next year. In addition, Novartis is testing Fabhalta in several other ongoing programs, including for anti-neutrophil cytoplasmic antibody (ANCA)–associated vasculitis, intermediate age-related macular degeneration, pediatric C3 glomerulopathy, paroxysmal nocturnal hemoglobinuria and IgA nephropathy. The fresh discontinuation also doesn’t mean that Novartis is giving up on lupus nephritis (LN). “Novartis remains committed to advancing LN research,” the spokesperson told BioSpace , pointing to the company’s ongoing Phase 3 study for the investigational monoclonal antibody ianalumab, for which data are expected in 2027, according to the Q2 report. Fabhalta’s mid-stage study shuttering comes after Novartis in September stopped a mid-stage study for its TREM2 stabilizer lifonebart for amyotrophic lateral sclerosis—the pharma’s fourth trial stumble in just a month’s time. The pharma experienced high-pro setbacks for the antibody-oligonucleotide conjugate del-desiran , the antisense therapy pelacarsen and the CAR T cell therapy rapcabtagene autoleucel . Neuroscience Novartis’ $12B Avidity acquisition hits Phase 3 speedbump as dystrophy drug disappoints While Novartis’ antibody-oligonucleotide conjugate failed to significantly improve hand function in patients with myotonic dystrophy type 1, the asset nevertheless showed signs of clinical activity in secondary and exploratory measures. September 8, 2026 · 2 min read · Tristan Manalac Read more Cardiovascular disease Novartis’ cholesterol-lowering medicine fails Phase 3, clouding Lp(a) horizons The failure of Novartis’ pelacarsen to translate its Lp(a)-lowering effects to clinical benefits “marks a meaningful setback” for other companies taking a similar approach to cardiovascular risk, analysts say—including Eli Lilly and Amgen. September 8, 2026 · 2 min read · Tristan Manalac Read more CAR-T Novartis pauses CAR T studies due to 3 deaths; BMS follows suit after safety events Novartis suspended multiple studies of a CAR T candidate after three patients who received the therapy suffered fatal cases of immune effector cell-associated hemophagocytic syndrome. BMS, meanwhile, paused some CAR T trials after separately observing “transient and reversible inflammatory events” with its therapy. September 1, 2026 · 3 min read · Tristan Manalac Read more