Aqvesme

Aqvesme is used to treat anemia in adults with alpha- or beta-thalassemia. It is an oral tablet taken twice daily regardless of whether the patient requires regular blood transfusions.

Aqvesme gained FDA approval on December 23, 2025.

Aqvesme is only available through a restricted program called the Aqvesme REMS because of the risk of serious liver damage.

• Prescribers must be certified by enrolling in the REMS and completing training.
• Prescribers must counsel patients receiving Aqvesme about the risk of hepatocellular injury.
• Prescribers must monitor liver tests (including ALT, AST, alkaline phosphatase, total bilirubin with fractionation, and other tests as clinically indicated) to determine if the patient is appropriate to receive Aqvesme treatment.
• Patients must enroll in the REMS and comply with the monitoring requirements.
• Pharmacies must be certified by enrolling in the REMS and must only dispense to patients who are authorized to receive Aqvesme.

Further information is available at www.aqvesmerems.com or 1-800-625-9951.

Aqvesme is taken 2 times a day.

• May be taken with or without food.
• Swallow the tablets whole; do not split, crush, chew, or dissolve.

Do not take Aqvesme if you have cirrhosis (Child-Pugh Class A, B, or C).

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

• Have cirrhosis or any liver problems (Aqvesme should not be used if you have cirrhosis)
• Take any other medications, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements
• Have had liver problems in the past
• Take hormonal birth control. Aqvesme may make hormonal birth control work less well. Use a different type of birth control or an additional nonhormonal method (such as condoms) during treatment and for 28 days after stopping Aqvesme.
• Are pregnant or planning to become pregnant. It is unknown if Aqvesme will affect an unborn baby
• Are breastfeeding or planning to breastfeed. It is unknown if Aqvesme passes into breastmilk.

Most common side effects

The most common side effects of Aqvesme are headache and insomnia (sleeplessness).

Serious side effects and warnings

Serious Liver Injury

Aqvesme can cause serious liver injury, typically within the first 6 months of treatment, and should not be used in patients with cirrhosis. Your healthcare provider will monitor your liver function tests (ALT, AST, alkaline phosphatase, bilirubin) at baseline, every 4 weeks for 24 weeks, then as needed. They will discontinue Aqvesme immediately if liver injury is suspected.

In clinical trials, five patients developed liver injury with peak ALT levels exceeding 5 times the upper limit of normal; two required hospitalization. All cases improved after stopping treatment.

Report symptoms of liver injury to your healthcare provider immediately, which may include:

• Loss of appetite
• Nausea
• Vomiting
• Right upper abdominal pain
• Yellowing of skin or eyes (jaundice)
• Dark urine.

Aqvesme is only available through a restricted REMS program.

To report suspected side effects, contact Agios Pharmaceuticals, Inc. at 1-833-228-8474 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Aqvesme works by enhancing the activity of pyruvate kinase (PK), an enzyme that improves red blood cell energy and survival. This leads to a greater number of healthier RBCs circulating in the body.

Aqvesme belongs to the drug class called pyruvate kinase activators.

Adult Dose of Aqvesme for Alpha- or Beta-Thalassemia

• 100 mg twice daily.

If you miss a dose of Aqvesme by:

• 4 hours or less, take the scheduled dose as soon as possible
• More than 4 hours, do not take a replacement dose. Wait until the next scheduled dose.

Tell your healthcare provider about all the medications you take, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements. Aqvesme interacts with many medications and some combinations should be avoided. Especially tell your healthcare provider if you take:

• Strong CYP3A inhibitors (e.g., ketoconazole, clarithromycin, ritonavir)
• Moderate CYP3A inhibitors (e.g., erythromycin, fluconazole, diltiazem)
• Strong CYP3A inducers (e.g., rifampin, phenytoin, carbamazepine, St. John's Wort)
• Hormonal birth control pills and other sensitive CYP3A medications with narrow safety margins
• Moderate CYP3A inducers (e.g., efavirenz, modafinil). Your doctor may need to adjust doses
• Certain blood thinners, seizure medications, and cholesterol drugs. Your doctor will monitor their effectiveness
• Digoxin and similar medications. Your doctor will watch for side effects

Always check with your healthcare provider before starting or stopping any medication while taking Aqvesme.

Store Aqvesme at room temperature between 20°C to 25°C (68°F to 77°F).

Store the blister wallets in the original carton until use.

Manufactured for and distributed by Agios Pharmaceuticals, Inc. Cambridge, MA 02139.