In adults
AQVESME is the ONLY treatment for anemia in BOTH non–transfusion-dependent (NTD) and transfusion-dependent (TD), alpha- or beta-thalassemia that1:
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Significantly increases hemoglobin (Hb) in NTD thalassemia*
Significantly reduces fatigue in NTD thalassemia†
Significantly reduces transfusion burden in TD thalassemia‡
Learn more about the FDA approval of AQVESME for thalassemia
*ENERGIZE is a randomized, double-blind, placebo-controlled, phase 3 clinical trial in participants with NTD ɑ- or β-thalassemia (N=194) that demonstrated 42.3% of participants on AQVESME achieved an Hb response (≥1 g/dL increase from baseline in average Hb from week 12 through week 24) vs 1.6% of participants on placebo (P<0.0001).1,2
†In ENERGIZE, participants on AQVESME achieved a 4.85-point increase from baseline in average FACIT-Fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue Scale) score from week 12 through week 24 vs an average 1.46-point increase for participants on placebo (P=0.0026).1
‡ENERGIZE-T is a randomized, double-blind, placebo-controlled, phase 3 trial in participants with TD ɑ- or β-thalassemia (N=258) that demonstrated 30.4% of participants on AQVESME achieved a transfusion reduction response (≥50% and ≥2-unit reduction in transfused red blood cell [RBC] units from baseline in any consecutive 12 weeks through week 48) vs 12.6% of participants on placebo (P=0.0003).1,3
Getting Started With AQVESME
When starting treatment with AQVESME, myAgios® Patient Support Services is here to help. myAgios is a customized support program for people living with thalassemia, designed to provide personalized education and treatment support.
AQVESME is only available through the AQVESME Risk Evaluation and Mitigation Strategy (REMS). Healthcare providers, patients, and pharmacies must complete the REMS enrollment process before treatment can begin. AQVESME REMS forms and guides will be available soon. For questions, call myAgios at 1-877-77-AGIOS (1-877-772-4467).
3 simple steps to help start treatment promptly:
Once the healthcare provider (HCP) and patient have reviewed AQVESME clinical and REMS information and decided that AQVESME is right for the patient, these are the steps to start treatment promptly:
HCP and patient complete and sign the AQVESME Start Form and the HCP completes one-time HCP AQVESME REMS certification
The AQVESME Start Form enrolls the patient in myAgios Patient Support Services. The myAgios team can assist HCPs and patients in determining insurance coverage and exploring financial assistance options.
Complete pre-treatment liver test
The pre-treatment liver test (blood test) must be completed within 4 weeks of the first prescription being filled. The myAgios team will work with the HCP and patient to determine the ideal timing to complete the required pre-treatment liver test based on status of insurance determination.
HCP enrolls patient in AQVESME REMS
To enroll, HCP and patient must complete and sign the AQVESME REMS Patient Enrollment Form, including documentation of patient’s pre-treatment liver test and confirmation that the patient is appropriate for AQVESME.
The myAgios team will ensure that AQVESME is delivered directly to the patient's home through an exclusive REMS-certified Specialty Pharmacy
myAgios: A customized support program for people living with thalassemia
The dedicated team at myAgios is made up of Agios Clinical Educators (ACEs), who deliver personalized education, and Patient Support Managers (PSMs), who provide customized treatment support.
myAgios offers help with:
- Disease education and support in understanding treatment options
- Accessing AQVESME and understanding insurance coverage
- Exploring ways to make AQVESME more affordable
- Ongoing treatment support for people who have been prescribed AQVESME
- Resources for living with thalassemia and community connections
Call 1-877-77-AGIOS (1-877-772-4467) Mon-Fri, 8 AM to 8 PM ET to connect with myAgios today.
AQVESME Educational Programs
FOR PROVIDERS
Sign up for Healthcare Professional (HCP) educational programs
Curious about AQVESME for thalassemia? Sign up for upcoming webinars to learn more about AQVESME efficacy and safety data.
FOR PEOPLE LIVING WITH THALASSEMIA
Register for educational programs for people with thalassemia
Interested in learning more about treatment with AQVESME? Hear from a thalassemia expert and connect with others living with thalassemia at one of our in-person or online programs.
Connect with us to learn more about AQVESME
Please complete the form below for more information about AQVESME, upcoming events, and resources.
You can also call 1-877-77-AGIOS (1-877-772-4467) Mon-Fri, 8 AM to 8 PM ET to get started with myAgios.
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References: 1. AQVESME. Prescribing Information. Agios Pharmaceuticals, Inc.; 2025. 2. Taher AT, Al-Samkari H, Aydinok Y, et al; ENERGIZE investigators. Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial. Lancet. 2025;406(10498):33-42. doi:10.1016/S0140-6736(25)00635-X 3. Cappellini MD, Sheth S, Taher AT, et al. ENERGIZE-T: a global, phase 3, double-blind, randomized, placebo-controlled study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia. Blood. 2024;144(suppl 1):409-411. doi:10.1182/blood-2024-200867
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.