Here is information on the latest US FDA approvals, the week of March 25 – March 29, 2024
Winrevair for PAH
The FDA has approved sotatercept-csrk (Winrevair) for the treatment of adults with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group 1) to increase exercise capacity, improve WHO functional class (FC), and reduce the risk of clinical worsening events. Sotatercept represents a new class of therapy as the first FDA-approved activin signaling inhibitor for PAH, which works by improving the balance between pro- and anti-proliferative signaling to regulate vascular cell proliferation underlying PAH. PAH is a rare, progressive condition marked by the thickening and narrowing of the blood vessels in the lungs, leading to increased strain on the heart. The approval was based on the Phase 3 STELLAR trial, which demonstrated that adding sotatercept to background PAH therapy significantly increased the six-minute walk distance by 41 meters at Week 24, improved multiple secondary outcome measures, and reduced the risk of death from any cause or PAH clinical worsening events by 84% compared to background therapy alone. The approval of sotatercept-csrk was granted to Merck.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761363s000lbl.pdf
Vafseo for CKD
The FDA has approved vadadustat (Vafseo) for the treatment of anemia due to chronic kidney disease (CKD) in adult patients on dialysis. This approval marks vadadustat as a novel treatment option in the landscape of CKD management, specifically targeting those who have been on dialysis for at least three months. Vadadustat, a once-daily oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor, functions by activating the body’s response to low oxygen levels, stimulating the endogenous production of erythropoietin, thereby managing anemia. The approval of vadadustat for the treatment of anemia due to CKD in adults who have been receiving dialysis for at least three months is based on efficacy and safety data from the INNO2VATE program and an assessment of post marketing safety data from Japan where VAFSEO was launched in August 2020. The approval of vadadustat was granted to Akebia Therapeutics.
https://www.prnewswire.com/news-releases/akebia-receives-fda-approval-of-vafseo-vadadustat-tablets-for-the-treatment-of-anemia-due-to-chronic-kidney-disease-in-adult-patients-on-dialysis-302101854.html
Ultomiris for NMOSD
The FDA has approved ravulizumab-cwvz (Ultomiris) for the treatment of patients with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). Ravulizumab-cwvz, a terminal complement C5 inhibitor require infusions only once every two months compared to the more frequent dosing schedules of some other treatments. Neuromyelitis optica spectrum disorder (NMOSD) is an autoimmune condition characterized by severe attacks affecting the optic nerves and spinal cord, often leading to blindness and paralysis. The role of complement inhibition in preventing NMOSD relapses highlights the critical nature of targeting the complement system in treatment strategies. The approval of ravulizumab-cwvz was supported by the Phase 3 CHAMPION-NMOSD study (NCT04201262), which demonstrated no relapses in 58 NMOSD patients over a median treatment duration of 73 weeks. This study’s findings are particularly notable for indicating a 98.6% reduction in relapse risk compared to placebo, with patients receiving ravulizumab achieving a 100% relapse-free status at 48 weeks versus 63% of those on placebo. The approval of ravulizumab-cwvz was granted to Alexion.
https://www.neurologylive.com/view/fda-approves-alexion-ravulizumab-nmosd
Evolut FX+ for TAVR
The FDA has approved Evolut™ FX+ transcatheter aortic valve replacement (TAVR) system for the treatment of symptomatic severe aortic stenosis. This system is an evolution of the existing Evolut TAVR platform, with larger windows that enhance catheter maneuverability for coronary artery access across diverse patient anatomies without compromising valve effectiveness or durability. Severe aortic stenosis, a condition characterized by the stiffening and thickening of the aortic valve leaflets, forces the heart to work harder, significantly impacting patient quality of life and potentially leading to heart failure if untreated. Medtronic’s Evolut FX+ TAVR system is now available for patients across all risk categories for aortic stenosis in the US, with a commercial rollout expected in spring 2024.
Vemlidy for Pediatric HBV
The FDA has approved tenofovir alafenamide (Vemlidy) for the treatment of chronic hepatitis B virus (HBV) infection in pediatric patients as young as 6 years of age and weighing at least 25 kg with compensated liver disease. Vemlidy was initially approved by the FDA in 2016 as a once-daily treatment for adults with chronic HBV infection with compensated liver disease. Subsequently, in 2022, the FDA extended the approval to include pediatric patients 12 years of age and older with compensated liver disease. Vemlidy operates as a targeted prodrug of tenofovir, recommended in certain guidelines as a preferred or first-line treatment for adults with chronic HBV with compensated liver disease. Chronic hepatitis B can lead to severe liver complications, including cirrhosis and liver cancer, if left untreated. The approval for this indication was based on findings from the Phase 2 clinical trial 1092, demonstrating the efficacy and safety of tenofovir alafenamide in a pediatric population. This expanded indication offers a new therapeutic option for younger children affected by this chronic disease, reflecting ongoing efforts to address treatment needs across diverse patient populations. The approval of Vemlidy for this younger age group was granted to Gilead Sciences.
https://www.gilead.com/~/media/files/pdfs/medicines/liver-disease/vemlidy/vemlidy_pi.pdf?la=en
Pemgarda for COVID
The FDA has approved Pemgarda, a monoclonal antibody, for preventive use in immunocompromised individuals aged 12 and older who are not currently infected with the virus that causes COVID-19 and have not been recently exposed. This agent operates by preventing the virus from attaching to human cells and is administered through an intravenous (IV) route. It is important to note that Pemgarda cannot be administered within 2 weeks of receiving a COVID-19 vaccine. Pemgarda has received emergency use authorization from the FDA, which has requested additional data from the ongoing CANOPY Phase 3 clinical trial to further study the drug. Preliminary results from this trial show that only 1% or fewer of the participants who received Pemgarda developed symptomatic COVID-19 within 90 days of treatment, compared to 5% in a control group with healthier immune systems. This indicates a potential significant benefit in preventing COVID-19 in people with compromised immune systems. The approval of Pemgarda was granted to Invivyd.
https://www.fda.gov/news-events/press-announcements/fda-roundup-march-22-2024