This is the third indication for Fabhalta after Novartis won FDA approval of the small molecule in paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
Novartis has secured a third FDA approval for Fabhalta—and the first approval for C3 glomerulopathy to reduce proteinuria, the company announced Friday.
C3 glomerulopathy (C3G) is a rare kidney disease that deposits protein fragments in the glomeruli, which can progress to end-stage kidney disease within 20 years of diagnosis. The average age at diagnosis is 23 years, according to Novartis.
The approval “was largely expected” after Novartis’ Phase III APPEAR-C3G study met the primary endpoint of proteinuria reduction from baseline at six months compared to placebo, William Blair analysts said in a Friday investor note.
Novartis won the first FDA approval for Fabhalta in December 2023 in paroxysmal nocturnal hemoglobinuria, a rare blood disease. The company then secured accelerated approval in 2024 for primary immunoglobulin A nephropathy, another kidney disease involving protein deposits.
APPEAR-C3G, a six-month double-blind study, tested a twice-daily oral formulation of Fabhalta followed by a six-month open label period where all patients got the small molecule treatment, including those in the previous period’s placebo arm. Results showed a reduction in proteinuria that Novartis said could be detected as early as 14 days after treatment and was sustained for one year on the drug.
Novartis reported no new safety signals. Side effects included the common cold and viral infections. Fabhalta can cause serious infections—including Streptococcus pneumoniae, which causes pneumonia, and Neisseria meningitidis, which causes meningitis—caused by “encapsulated bacteria,” and therefore will “only be available through a Risk Evaluation and Mitigation Strategy (REMS) that requires specific vaccinations,” according to the company.
“This approval of Fabhalta is historic for the entire C3G community as now, for the first time, we have a therapy that is believed to treat the underlying cause of the disease, providing the potential for a new standard of care for patients,” Carla Nester, co-investigator of the APPEAR-C3G trial, said in a statement.
The approval also marks another win for Novartis this week. On Wednesday, the company presented Phase III data at the Muscular Dystrophy Association’s Clinical & Scientific Conference showing that an intrathecal (IT) formulation of its spinal muscular atrophy gene therapy Zolgensma—approved in 2019 as an intravenous treatment for children under two years of age—appears to be effective in treating older SMA patients. The company plans to file for approval of the IT formulation in the first half of this year.
