Frequently asked questions (FAQs)

Get answers to common questions about AQVESME as well as helpful information about thalassemia.

Thalassemia FAQs

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Thalassemia is a rare, inherited blood disorder that causes your body to make less hemoglobin than usual. Hemoglobin is the protein in red blood cells (RBCs) that carries oxygen. When you don’t have enough healthy RBCs, your body doesn’t get enough oxygen, which can lead to serious complications.
There’s less healthy hemoglobin in people with thalassemia, which means less oxygen is carried throughout the body. This results in chronic anemia that can last throughout life.
Learn more about thalassemia
Thalassemia happens when there are changes to the genes that tell the body how to make hemoglobin. When these changes happen, hemoglobin is not made properly. This leads to fewer and more fragile red blood cells.
Learn more about the causes of thalassemia
There are 2 main types of thalassemia: alpha-thalassemia and beta-thalassemia. The type of thalassemia you have depends on which part of the hemoglobin protein is affected. Alpha-thalassemia is caused by changes in the alpha-globin genes, and beta-thalassemia is caused by changes in the beta-globin genes.
Learn more about alpha- and beta-thalassemia
Traditionally, thalassemia has been described as thalassemia major, thalassemia intermedia, thalassemia minor, and thalassemia trait, which refer to genetics and severity of anemia.
Learn more about thalassemia types
Doctors also may describe thalassemia as non–transfusion dependent (NTD) or transfusion dependent (TD). NTD thalassemia means you don’t require regular blood transfusions, but you may get them sometimes. TD thalassemia means you require regular lifetime blood transfusions for survival.
Learn more about NTD and TD thalassemia
The symptoms of thalassemia can be different for each person. Some symptoms of thalassemia include fatigue, weakness, shortness of breath, dizziness and fainting, paleness, and headaches. It’s important to be aware of your symptoms and share them with your care team.
Learn more about thalassemia symptoms

AQVESME FAQs

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In adults, AQVESME is the ONLY treatment for anemia in BOTH non–transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia.
Learn more about who can take AQVESME
AQVESME is a prescription medicine used to treat anemia (low red blood cells) in adults with alpha- or beta-thalassemia.
Learn more about AQVESME
AQVESME boosts the energy production inside red blood cells (RBCs) and is thought to strengthen RBCs for proper development and defend against early RBC breakdown.*
Learn more about how AQVESME is thought to work

*Based on early pre-clinical research in beta-thalassemia.

The efficacy and safety of AQVESME was studied in 2 clinical trials in people with alpha- or beta-thalassemia. The 2 clinical trials were called ENERGIZE and ENERGIZE-T. ENERGIZE included people who were non–transfusion dependent. ENERGIZE-T included people who were transfusion dependent.
Learn more about how AQVESME was studied
In the ENERGIZE clinical trial, AQVESME was shown to significantly increase hemoglobin and reduce fatigue.
See full study results

NTD=non–transfusion dependent.

In the ENERGIZE-T clinical trial, AQVESME was shown to significantly reduce transfusion burden. This may mean your transfusions are less frequent, or you may need fewer RBC units at each visit.
See full study results

TD=transfusion dependent.

The most common side effects include headache and trouble sleeping (insomnia).
Learn more about AQVESME safety
There is a potential risk of liver injury with AQVESME. Liver injury has happened in people with thalassemia within the first 6 months of treatment with AQVESME. Your healthcare provider will do blood tests to check your liver before you start treatment with AQVESME, every 4 weeks for the first 24 weeks of treatment, and as needed. Your healthcare provider may temporarily or permanently stop your treatment with AQVESME if you have abnormal liver blood tests.
Tell your healthcare provider right away if you develop any signs or symptoms of liver injury, including: yellowing of the skin or whites of the eyes (jaundice), dark-colored urine, pain in the upper right side of the stomach area, nausea, vomiting, or loss of appetite.
Your healthcare provider may temporarily or permanently stop your treatments with AQVESME if you have abnormal liver blood test results.
See more safety information
AQVESME is only available through AQVESME REMS, a program designed to inform you of the potential risk of liver injury and to monitor your liver function. REMS programs are required by the Food and Drug Administration (FDA) for certain medicines to help manage the risk of serious side effects.
Your doctor will enroll you in AQVESME REMS to regularly monitor your liver function. They will provide you information about the potential risk of liver injury and about regular liver monitoring requirements. Blood tests will be done:
  • Before you start treatment
  • Every 4 weeks for the first 24 weeks, and as needed
Learn more about AQVESME REMS
AQVESME is 1 tablet that is taken twice a day. Take AQVESME with or without food. Swallow AQVESME whole (do not split, chew, crush, or dissolve the tablets). Take AQVESME as directed by your healthcare provider.
See more information about taking AQVESME
Once you and your doctor have decided that AQVESME is right for you, there are 3 key actions to help you start treatment as soon as possible.
Learn how to get started
Your doctor is your most important resource when it comes to your thalassemia care. You can also connect with an Agios Clinical Educator (ACE) to learn more about AQVESME. Call 1-877-77-AGIOS (1-877-772-4467) Mon-Fri, 8 AM to 8 PM ET. An ACE works closely with you, your family, and your healthcare team to provide one-on-one disease education and personalized support.
Agios also offers free educational events both online and in person where you can learn more about AQVESME and connect with others living with thalassemia.
Learn more about these programs

ACEs do not provide medical advice. For medical advice or treatment-related questions, please talk to your healthcare team.

myAgios Patient Support Services is a customized support program for people living with thalassemia designed to provide personalized education and treatment support. myAgios offers help with:
  • Disease education and support in understanding treatment options
  • Resources for living with thalassemia
  • Ongoing treatment support for people who have been prescribed AQVESME
  • Accessing AQVESME and understanding insurance coverage
  • Exploring ways to make AQVESME more affordable
You can connect with an Agios Clinical Educator (ACE) to learn more about AQVESME. Call 1-877-77-AGIOS (1-877-772-4467) Mon-Fri, 8 AM to 8 PM ET.
Learn more about myAgios

ACEs do not provide medical advice. For medical advice or treatment-related questions, please talk to your healthcare team.

NTD=non-transfusion dependent; TD=transfusion dependent.

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IMPORTANT SAFETY INFORMATION

AQVESME may cause serious side effects, including:

  • Liver injury. AQVESME can cause serious liver injury. Liver injury has happened in people with thalassemia within the first 6 months of treatment with AQVESME. Your healthcare provider will do blood tests to check your liver before you start treatment with AQVESME, every 4 weeks for the first 24 weeks of treatment, and as needed. Your healthcare provider may temporarily or permanently stop your treatment with AQVESME if you have abnormal liver blood tests.

    Tell your healthcare provider right away if you develop any new or worsening signs or symptoms of liver problems, including:

    • loss of appetite
    • nausea
    • pain in the upper right side of your stomach area
    • vomiting
    • yellowing of the skin and white part of your eyes (jaundice)
    • dark-colored urine

In clinical studies of AQVESME, 2 of 301 people (0.66%) treated with AQVESME experienced adverse reactions suggestive of liver injury. Three additional people experienced adverse reactions suggestive of liver injury during the open-label extension periods, after switching from placebo to AQVESME. Of the 5 people, two had serious liver injury requiring hospitalization, including 1 who developed jaundice. Another developed jaundice without requiring hospitalization. All 5 people discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation.

Because of the risk for liver injury, AQVESME is only available through a restricted access program called the AQVESME Risk Evaluation and Mitigation Strategy (REMS).

Before taking AQVESME, tell your healthcare provider about all of your medical conditions, including if you:

  • have liver problems, such as cirrhosis
  • are pregnant or plan to become pregnant. It is not known if AQVESME will harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with AQVESME.
  • are breastfeeding or plan to breastfeed. It is not known if AQVESME passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with AQVESME.

Tell your healthcare provider about all the medicines that you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Make sure to tell your healthcare provider if you take or use hormonal birth control (contraceptives). If you take or use hormonal birth control (except for intrauterine systems containing levonorgestrel), it may not work as well during treatment with AQVESME. Use a different type of birth control or use an additional nonhormonal birth control method (such as condoms) during treatment with AQVESME and for 28 days after stopping treatment with AQVESME. AQVESME and certain other medicines may affect each other and cause side effects. AQVESME may affect the way other medicines work, and other medicines may affect the way AQVESME works.

The most common side effects of AQVESME in patients:

  • headache
  • insomnia

These are not all of the possible side effects of AQVESME. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What is AQVESME?

AQVESME is a prescription medicine used to treat anemia (low red blood cells) in adults with alpha- or beta-thalassemia. It is not known if AQVESME is safe and effective in children.

Please see full Prescribing Information, including Medication Guide.

IMPORTANT SAFETY INFORMATION

AQVESME may cause serious side effects, including:

  • Liver injury. AQVESME can cause serious liver injury. Liver injury has happened in people with thalassemia within the first 6 months of treatment with AQVESME. Your healthcare provider will do blood tests to check your liver before you start treatment with AQVESME, every 4 weeks for the first 24 weeks of treatment, and as needed. Your healthcare provider may temporarily or permanently stop your treatment with AQVESME if you have abnormal liver blood tests.

    Tell your healthcare provider right away if you develop any new or worsening signs or symptoms of liver problems, including:

    • loss of appetite
    • nausea
    • pain in the upper right side of your stomach area
    • vomiting
    • yellowing of the skin and white part of your eyes (jaundice)
    • dark-colored urine

In clinical studies of AQVESME, 2 of 301 people (0.66%) treated with AQVESME experienced adverse reactions suggestive of liver injury. Three additional people experienced adverse reactions suggestive of liver injury during the open-label extension periods, after switching from placebo to AQVESME. Of the 5 people, two had serious liver injury requiring hospitalization, including 1 who developed jaundice. Another developed jaundice without requiring hospitalization. All 5 people discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation.

Because of the risk for liver injury, AQVESME is only available through a restricted access program called the AQVESME Risk Evaluation and Mitigation Strategy (REMS).

Before taking AQVESME, tell your healthcare provider about all of your medical conditions, including if you:

  • have liver problems, such as cirrhosis
  • are pregnant or plan to become pregnant. It is not known if AQVESME will harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think that you are pregnant during treatment with AQVESME.
  • are breastfeeding or plan to breastfeed. It is not known if AQVESME passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with AQVESME.

Tell your healthcare provider about all the medicines that you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Make sure to tell your healthcare provider if you take or use hormonal birth control (contraceptives). If you take or use hormonal birth control (except for intrauterine systems containing levonorgestrel), it may not work as well during treatment with AQVESME. Use a different type of birth control or use an additional nonhormonal birth control method (such as condoms) during treatment with AQVESME and for 28 days after stopping treatment with AQVESME. AQVESME and certain other medicines may affect each other and cause side effects. AQVESME may affect the way other medicines work, and other medicines may affect the way AQVESME works.

The most common side effects of AQVESME in patients:

  • headache
  • insomnia

These are not all of the possible side effects of AQVESME. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What is AQVESME?

AQVESME is a prescription medicine used to treat anemia (low red blood cells) in adults with alpha- or beta-thalassemia. It is not known if AQVESME is safe and effective in children.

Please see full Prescribing Information, including Medication Guide.