Novartis announces that it has received US FDA approval for its Fabhalta (iptacopan), as the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH), a chronic and rare blood disorder.

This approval is based on the APPLY-PNH trial in adults with HPN and anemia despite prior anti-C5 therapy, and is supported by the APPOINT-PNH study in complement inhibitor-naïve patients.

Significant unmet needs remain in HPN; despite anti-C5 treatment, a large proportion of patients may remain anemic and dependent on blood transfusions", stresses the Swiss pharmaceutical company.

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