Novartis OKd for paroxysmal nocturnal hemoglobinuria treatment

 

• Approval based on APPLY-PNH trial in adults with PNH and anemia despite prior anti-C5 treatment, and supported by the APPOINT-PNH study in complement inhibitor-naïve patients^1-5
   
• In APPLY-PNH, patients who switched to Fabhalta experienced superior increases of hemoglobin levels ≥ 2 g/dL (82.3% vs. 0%) and hemoglobin level ≥ 12 g/dL (67.7% vs. 0%), both in the absence of red blood cell transfusions, vs. patients who continued on anti-C5 treatment^1,2  
   
• 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^1,6
   
• Significant unmet need remains in PNH, a chronic and rare blood disorder; despite anti-C5 therapy, a large proportion of patients can remain anemic and dependent on blood transfusions^7,8
   
• Late-stage Fabhalta development program ongoing in multiple complement-mediated conditions

https://www.biospace.com/article/releases/novartis-receives-fda-approval-for-fabhalta-iptacopan-offering-superior-hemoglobin-improvement-in-the-absence-of-transfusions-as-the-first-oral-monotherapy-for-adults-with-pnh/