March 22,NovartisAnnouncementThe Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending the granting ofFabhalta（iptacopan) Marketing Authorization,For patients with hemolytic anemiaAdult Paroxysmal Nocturnal Hemoglobinuria (PNH).FabhaltaIt is a first-in-class, oral inhibitor targeting the complement alternative pathway B factor developed by Novartis. The product acts upstream of the terminal C5 pathway, simultaneously controlling intravascular hemolysis and extravascular hemolysis, addressing the shortcomings of anti-C5 antibodies while providing patients with an oral monotherapy option. In December 2023,FabhAlta Receives FDA Approval for Market Launch,UseFor adult PNThe treatment of Patient H has becomeThe first drug approved by the FDA for oral monotherapy treatment of adult PNH.
CHMP positive recommendation is mainly based onPhase IIIAPPLY-PNH andData from the APPOINT-PNH Two Studies。APPLY-PNHResearchTo evaluate the efficacy and safety of iptacopan monotherapy (200 mg) administered orally twice daily for the treatment of PNH.And proveReceived stable treatment in the 6 months prior to randomizationComplementResidual anemia still occurs with C5 treatment regimenIn PNH patients,Iptacopan vs. C5 monoclonal antibody therapy (eculizumabOrravulizumab) Superiority.
Key results showed that, in patients who continued to take iptacopan (200mg twice daily) after prior C5 complement therapy and did not require transfusions over 24 weeks, 82.3% achieved a ≥2g/dL increase in hemoglobin levels from baseline, compared toC5 monoclonal antibodies (2.0%) have unifiedStatistical significance and clinical relevance were increased, reaching the primary endpoint.At the same time, compared with the C5 monoclonal antibody, the proportion of patients in the iptcopan group who did not require a blood transfusion and reached a hemoglobin level of ≥12 g/dL at 24 weeks was 67%..7% (P<0.0001), achieving another primary endpoint. Additionally, patients previously treated with C5 complement therapy had a transfusion avoidance rate of 94.8% when continuing treatment with iptacopan, compared to 25.9% in the C5 complement therapy group (P<0.0001).APPOINT-PNH aims to evaluate the efficacy and safety of iptacopan monotherapy in adult PNH patients who have not received prior treatment with complement inhibitors, including anti-C5 antibodies.The results showed,92.2% of patients achieved hemoglobin levels without red blood cell transfusion (RBCT).Increased from baseline ≥2g/dl。Secondary endpoints showed clinically meaningful improvements, with 97.6% of patients avoiding transfusions and 62.8% of patients achieving sustained hemoglobin levels without red blood cell transfusions.≥12g/dl。In two studies, iptacopan was also shown to control intravascular hemolysis, with mean LDH levels controlled at<1.5x upper limit of normal, etc.According to CHMP recommendation for approvalFabhaltaFor adult PNH patients with hemolytic anemia, the European Commission (EC) is expected to make a final decision within two months.Copyright © 2024 PHARMCUBE. All Rights Reserved.
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