Novartis Secures FDA Approval for Fabhalta in Rare Kidney Disease, Marking Third Indication

Novartis has achieved a significant milestone with the U.S. Food and Drug Administration's (FDA) approval of Fabhalta for the treatment of C3 glomerulopathy (C3G), a rare kidney disease. This marks the third indication for the small molecule therapy, following previous approvals for paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.

Breakthrough in C3 Glomerulopathy Treatment

C3G is a rare kidney condition characterized by the deposition of protein fragments in the glomeruli, which can lead to end-stage kidney disease within two decades of diagnosis. The average age at diagnosis is 23 years. Fabhalta, now approved to reduce proteinuria in C3G patients, represents the first therapy believed to address the underlying cause of the disease.

Dr. Carla Nester, co-investigator of the APPEAR-C3G trial, hailed the approval as "historic for the entire C3G community," noting that it offers "the potential for a new standard of care for patients."

Clinical Trial Results and Safety Profile

The FDA's decision was based on the Phase III APPEAR-C3G study, which demonstrated Fabhalta's efficacy in reducing proteinuria from baseline at six months compared to placebo. Key findings include:

• Proteinuria reduction detectable as early as 14 days after treatment initiation
• Sustained effect for one year on the drug
• No new safety signals reported

Common side effects included the common cold and viral infections. However, Fabhalta can cause serious infections, particularly those caused by encapsulated bacteria such as Streptococcus pneumoniae and Neisseria meningitidis. As a result, the drug will only be available through a Risk Evaluation and Mitigation Strategy (REMS) that mandates specific vaccinations.

Novartis' Expanding Therapeutic Portfolio

This approval adds to Novartis' recent successes in the pharmaceutical arena. Earlier this week, the company presented promising Phase III data for an intrathecal formulation of its spinal muscular atrophy gene therapy, Zolgensma, at the Muscular Dystrophy Association's Clinical & Scientific Conference. The new formulation shows potential efficacy in treating older SMA patients, expanding upon the intravenous treatment currently approved for children under two years of age.

Novartis plans to file for approval of the intrathecal formulation of Zolgensma in the first half of this year, further solidifying its position in the rare disease treatment landscape.