Novartis' Rare Blood Disorder Therapy Scores FDA Approval

The FDA approved Novartis AG's NVS Fabhalta (iptacopan) as the first oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria (PNH). 

PNH is an acquired (not inherited) disorder that leads to premature death and impaired production of blood cells. It is estimated that approximately 10-20 people per million worldwide live with PNH.

Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis [IVH and EVH]). 

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

The FDA approval is based on the Phase III APPLY-PNH trial in patients with residual anemia (hemoglobin < 10 g/dL) despite prior anti-C5 treatment who switched to Fabhalta, which demonstrated superiority in hemoglobin improvement in the absence of RBC transfusions and transfusion avoidance rate over patients who stayed on anti-C5 treatments.

Discovered and developed by Novartis, Fabhalta is expected to be available in the United States in December. Additional regulatory filings and reviews for Fabhalta in PNH are underway worldwide.

Price Action: NVS shares closed at $96.98 on Tuesday.