Winrevair for PAH, Vafseo for CKD, Ultomiris for NMOSD, Evolut FX+ for TAVR, Vemlidy for Pediatric HBV, Pemgarda for COVID

New FDA Approvals

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• Winrevair for PAH: Sotatercept-csrk (Winrevair) is FDA-approved for treating adults with pulmonary arterial hypertension (PAH), enhancing exercise capacity, improving WHO functional class, and reducing clinical worsening events. It's the first FDA-approved activin signaling inhibitor for PAH, addressing the imbalance in vascular cell proliferation underlying the condition. The approval is based on the Phase 3 STELLAR trial, demonstrating significant improvements in walk distance and reduced risk of clinical worsening events [1].
• Vafseo for CKD: Vadadustat (Vafseo) is approved for managing anemia due to chronic kidney disease (CKD) in adult dialysis patients. As an oral HIF-PH inhibitor, it stimulates endogenous erythropoietin production, offering a novel approach to anemia management. Approval is supported by efficacy and safety data from the INNO2VATE program and post-marketing safety data from Japan [2].
• Ultomiris for NMOSD: Ravulizumab-cwvz (Ultomiris) is FDA-approved for treating neuromyelitis optica spectrum disorder (NMOSD) in patients with anti-AQP4 antibodies. It's a terminal complement C5 inhibitor administered once every two months, demonstrating efficacy in preventing relapses. Approval is based on the Phase 3 CHAMPION-NMOSD study, showing significant reductions in relapse risk compared to placebo [3].
• Evolut FX+ for TAVR: The Evolut™ FX+ transcatheter aortic valve replacement (TAVR) system is FDA-approved for treating symptomatic severe aortic stenosis. It features enhancements for improved catheter maneuverability without compromising effectiveness. The approval expands treatment options for patients across all risk categories [4].
• Vemlidy for Pediatric HBV: Tenofovir alafenamide (Vemlidy) is FDA-approved for treating chronic hepatitis B virus (HBV) infection in pediatric patients aged 6 years and older with compensated liver disease. It's a preferred or first-line treatment option, addressing a significant medical need. Approval is based on the Phase 2 clinical trial 1092, demonstrating efficacy and safety in this patient population [5].
• Pemgarda for COVID: Pemgarda, a monoclonal antibody, is FDA-approved for preventive use in immunocompromised individuals aged 12 and older against COVID-19. It prevents virus attachment and has shown promising results in reducing symptomatic COVID-19 cases. Approval is based on emergency use authorization and preliminary data from the CANOPY Phase 3 clinical trial [6].