Atypical hemolytic uremic syndrome treatment with oral complement inhibitor therapy and rare disease clinical trials
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Novartis Receives FDA Approval for Fabhalta in Atypical Hemolytic Uremic Syndrome

Novartis announces FDA approval of Fabhalta (iptacopan) for adults with atypical hemolytic uremic syndrome, providing a new oral treatment option for this rare disease.

Novartis today announced that the FDA has approved Fabhalta (iptacopan) for the treatment of adults with atypical hemolytic uremic syndrome (aHUS). The approval was based on results from a Phase 3 trial that demonstrated a statistically significant improvement in complete thrombotic microangiopathy (TMA) response compared to placebo. Fabhalta is an oral proximal complement inhibitor targeting Factor B of the alternative complement pathway. This approval provides patients with aHUS a new oral treatment option, potentially offering a more convenient alternative to intravenous complement inhibitors.

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