{"id":1006,"date":"2026-08-27T22:52:51","date_gmt":"2026-08-27T22:52:51","guid":{"rendered":"https:\/\/noimfine.com\/sph06\/?p=1006"},"modified":"2026-09-27T22:54:08","modified_gmt":"2026-09-27T22:54:08","slug":"aqvesme-fda-approves-first-pill-for-treating-anemia-in-adults-with-alpha-or-beta-thalassemia-2","status":"publish","type":"post","link":"https:\/\/noimfine.com\/sph06\/2026\/08\/27\/aqvesme-fda-approves-first-pill-for-treating-anemia-in-adults-with-alpha-or-beta-thalassemia-2\/","title":{"rendered":"Aqvesme: FDA Approves First Pill for Treating Anemia in Adults With Alpha- or Beta- Thalassemia"},"content":{"rendered":"<div><!--[--><!--&gt;--><\/p>\n<div>\n<div>\n<div>\n<div><\/div>\n<\/div>\n<\/div>\n<\/div>\n<div>\n<div>\n<section><a href=\"https:\/\/www.webmd.com\/drugs\/aqvesme-mitapivat\">Aqvesme (mitapivat)<\/a> is a new oral medicine approved by the FDA for treating anemia in adults with alpha- or beta-thalassemia, including those who need and those who don\u2019t require regular blood transfusions.<iframe src=\"https:\/\/www.youtube.com\/embed\/a9MDfJmmYCw\" width=\"560\" height=\"315\" frameborder=\"0\" allowfullscreen=\"allowfullscreen\"><\/iframe><\/p>\n<p>Thalassemia is a rare inherited blood disorder that affects how the body makes hemoglobin, a protein in the red blood cells (RBCs) that carries oxygen.<iframe src=\"https:\/\/www.youtube.com\/embed\/DnflkpzZxWM\" width=\"560\" height=\"315\" frameborder=\"0\" allowfullscreen=\"allowfullscreen\"><\/iframe><\/p>\n<p>Depending on which part of the hemoglobin structure (globin chain) is affected, thalassemia is categorized into two main types: alpha-thalassemia and beta-thalassemia.<iframe src=\"https:\/\/www.youtube.com\/embed\/7maxX41R_yA\" width=\"560\" height=\"315\" frameborder=\"0\" allowfullscreen=\"allowfullscreen\"><\/iframe><\/p>\n<p>Due to impaired hemoglobin production, people with thalassemia have fewer healthy RBCs with shorter life span than the healthy ones. These RBCs break down faster, leading to anemia with tiredness, weakness, and potentially serious long-term health problems. Some people with thalassemia need regular blood transfusions to manage their anemia (transfusion-dependent thalassemia), while others do not (non-transfusion-dependent thalassemia). Regardless of type, thalassemia requires lifelong care and monitoring because it can cause heart, liver, and blood vessel complications and significantly impact daily life. In the U.S., about 6,000 adults are living with thalassemia.<\/section>\n<section>Aqvesme works by boosting the activity of an enzyme called pyruvate kinase, which helps RBCs improve their energy balance and live longer, leading to a greater number of healthier RBCs circulating in the body. Aqvesme is the first FDA-approved treatment for anemia in both types of thalassemia, providing a new option that may help reduce the need for repeated blood transfusions. It also offers a treatment option to patients with non\u2011transfusion\u2011dependent thalassemia who previously had none.<\/section>\n<section>\n<h2>Why Was It Approved?<\/h2>\n<p>Aqvesme was approved after clinical studies showed it was well tolerated and effective in improving anemia, reducing the need for transfusions and enhancing quality of life in adults with non-transfusion-dependent or transfusion-dependent alpha- or beta-thalassemia. In a 48-week multicenter study involving 258 adults with transfusion-dependent alpha- or beta-thalassemia, participants were randomly assigned in a 2:1 ratio to receive either <a>an Aqvesme\u00a0<\/a>pill or a placebo twice daily. Results showed that Aqvesme significantly reduced the need for blood transfusions compared with placebo. About 30% of patients on Aqvesme versus 13% on placebo had a greater than 50% reduction in transfused RBC units in any consecutive 12-week period through week 48, and nearly 10% on Aqvesme versus 1% on placebo became transfusion-free for a period of eight or more consecutive weeks. The treatment also showed sustained reductions in transfused RBC units over longer periods and improved anemia. Aqvesme was generally well tolerated over the study period. The most common side effects were headache and trouble sleeping. In one of the clinical trials, a few people had liver-related side effects, including serious liver damage. Because of this, the FDA requires regular liver testing and limits access to the medicine through a special safety program called a Risk Evaluation and Mitigation Strategy (REMS).<\/section>\n<\/div>\n<div>\n<section>\n<h2>What Do I Need to Know?<\/h2>\n<p>Aqvesme comes as a tablet you take twice daily, with or without food. Swallow tablets whole without splitting, crushing, chewing, or dissolving them. If you miss a dose by four hours or less, take it as soon as possible. If it\u2019s been more than four hours, skip the missed dose and take your next dose at the usual time. Before starting Aqvesme, your health care provider will check your liver function. You\u2019ll need liver tests every four weeks for the first six months of treatment and even afterward if your health care provider considers them necessary. Inform your health care provider if you have liver disease or are undergoing treatment for any current liver problems. You should not take this medicine if you have liver damage or cirrhosis of a certain severity. Aqvesme may affect how other medicines work, including hormonal birth control pills and medicines that are processed by the liver. Be sure to tell your health care provider about all the medicines, over-the-counter products, vitamins, herbal remedies, and health supplements you take.<\/section>\n<section>Talk to your health care provider if you are pregnant, planning to become pregnant, breastfeeding, or planning to breastfeed as it is not known whether Aqvesme can harm your baby or pass into your breast milk. Contact your health care provider immediately if you experience loss of appetite, nausea or vomiting, pain on the right side of the belly, yellowing of the skin or eyes, or dark urine as these may be signs of liver problems that need prompt evaluation. Aqvesme is expected to be available in the U.S. starting late January 2026. However, it will only be available through certified health care providers and pharmacies that are part of the Aqvesme REMS program.<\/section>\n<\/div>\n<\/div>\n<p><!--&gt;<\/div>\n\n\n--><\/div>\n","protected":false},"excerpt":{"rendered":"<p>Aqvesme (mitapivat) is a new oral medicine approved by the FDA for treating anemia in adults with alpha- or beta-thalassemia, including those who need and those who don\u2019t require regular blood transfusions. Thalassemia is a rare inherited blood disorder that&#8230; <\/p>\n","protected":false},"author":2,"featured_media":528,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-1006","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"_links":{"self":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts\/1006","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/comments?post=1006"}],"version-history":[{"count":1,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts\/1006\/revisions"}],"predecessor-version":[{"id":1014,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts\/1006\/revisions\/1014"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/media\/528"}],"wp:attachment":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/media?parent=1006"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/categories?post=1006"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/tags?post=1006"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}