Novartis' Fabhalta Gets FDA Approval To Treat Paroxysmal Nocturnal Hemoglobinuria In Adults

Swiss drug major Novartis AG (NVS) Wednesday announced that the U.S. Food and Drug Administration or FDA has approved its Fabhalta (iptacopan) as the first oral monotherapy to treat adults with paroxysmal nocturnal hemoglobinuria or PNH.

Fabhalta, discovered and developed by Novartis, is now expected to be available in the United States in December.

Fabhalta, a prescription medicine, is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system. It provides comprehensive control of red blood cell or RBC destruction within and outside the blood vessels.

The FDA approval is based on the Phase III APPLY-PNH trial in patients with residual anemia despite prior anti-C5 treatment, and supported by the APPOINT-PNH study in complement inhibitor-naïve patients.

According to the company, Fabhalta, now available for both previously treated and treatment-naïve patients, is the only FDA-approved Factor B inhibitor of the immune system’s complement pathway, which drives complement-mediated hemolysis in PNH.

In clinical trials, treatment with Fabhalta increased hemoglobin levels in the majority of patients and in APPLY-PNH nearly all patients treated with Fabhalta did not receive blood transfusions.

Additional regulatory filings and reviews for Fabhalta in PNH are currently underway across the world.

Victor Bultó, President US, Novartis, said, “This new, effective oral medicine may mean that patients can reset their expectations of living with PNH, a chronic and life-altering blood disease. As Novartis continues to focus on conditions with unmet patient need, we are exploring the potential of Fabhalta in other complement-mediated diseases – with an ultimate goal to drive meaningful change for patients.”

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