AQVESME offers a demonstrated safety profile in both NTDT and TDT1

The safety of AQVESME was studied in 301 patients with thalassemia in two phase 3 trials1-3*

A table showing the percentage of common adverse reactions for AQVESME and placebo in both NTDT and TDT patients during the clinical trials
  • Most cases of headache and insomnia were mild (grade 1) to moderate (grade 2)4

HbC=hemoglobin C; HbE=hemoglobin E; HbH=hemoglobin H; HbS=hemoglobin S; NTDT=non–transfusion-dependent thalassemia; TDT=transfusion-dependent thalassemia.

*For patients with documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, H bE/β-thalassemia, or α-thalassemia/HbH disease), and excluding those who are homozygous or heterozygous HbS or HbC.1,2,4

†Included adverse reactions that occurred in at least 5% of patients in the AQVESME arm and at least 5% higher than the placebo arm.1

‡Insomnia (grouped preferred terms) includes: initial insomnia, middle insomnia, insomnia, and terminal insomnia.1

AQVESME REMS: supporting monitoring for potential risk of hepatocellular injury1

A Risk Evaluation and Mitigation Strategy (REMS) is required to monitor for potential risk of hepatocellular injury

  • In patients with thalassemia treated with AQVESME, adverse reactions suggestive of hepatocellular injury were observed within the first 6 months of treatment:
    • 2 of 301 (0.66%) patients during the double-blind period
    • 3 additional patients during the open-label extension after switching from placebo
  • 2 of the 5 events were serious (requiring hospitalization)
  • These reactions were characterized by peak elevations of ALT >5x upper limit of normal (ULN) with or without jaundice
  • All events improved upon treatment discontinuation

ALT=alanine aminotransferase.

Prescribe with confidence: enrolling in AQVESME REMS

  • AQVESME has a REMS due to the potential risk of hepatocellular injury. The goal of the AQVESME REMS is to help mitigate the risk of hepatocellular injury by monitoring liver function (ALT, AST, alkaline phosphatase, and total bilirubin with fractionation) in patients receiving AQVESME
  • Patient monitoring requirements include a pre-treatment liver test completed within 4 weeks of initiating therapy and monitoring liver function every 4 weeks during the first 24 weeks of treatment and as clinically indicated thereafter
  • Prescribers monitor liver function tests to determine the appropriateness of continuing therapy

Liver test monitoring schedule for the first 24 weeks of treatment

Timeline showing a required blood test before starting AQVESME, followed by tests every 4 weeks through week 24Timeline showing a required blood test before starting AQVESME, followed by tests every 4 weeks through week 24
  • Under the AQVESME REMS, only certified healthcare providers and pharmacies may prescribe and dispense AQVESME. Patients must be enrolled in the REMS program and comply with all program requirements to receive AQVESME

Learn more about AQVESME REMS at AQVESMEREMS.com

Download the AQVESME REMS Enrollment Overview Brochure for HCPs

AST=aspartate aminotransferase.

||AQVESME is one pill taken twice daily, supplied as a 28-day pack.1

Get your NTDT and TDT patients started on AQVESME

GETTING STARTED WITH AQVESME

Explore convenient oral dosing with AQVESME

DOSING AND ADMINISTRATION

References: 1. AQVESME. Prescribing Information. Agios Pharmaceuticals, Inc.; 2025. 2. Taher AT et al. Lancet. 2025;406(10498):33-42. doi:10.1016/S0140-6736(25)00635-X 3. Cappellini MD et al. Blood. 2024;144(suppl 1):409-411. doi:10.1182/blood-2024-200867 4. Data on file. Agios Pharmaceuticals, Inc.

IMPORTANT SAFETY INFORMATION

BOXED WARNING: HEPATOCELLULAR INJURY

AQVESME can cause serious hepatocellular injury. Measure liver laboratory tests (ALT, AST, alkaline phosphatase and total bilirubin with fractionation) at baseline and every 4 weeks for 24 weeks and then as clinically indicated. Avoid use of AQVESME in patients with cirrhosis. Discontinue AQVESME if hepatic injury is suspected.
Because of the risk of hepatocellular injury, AQVESME is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the AQVESME REMS.

WARNINGS AND PRECAUTIONS

Hepatocellular Injury

AQVESME can cause hepatocellular injury. Avoid use of AQVESME in patients with cirrhosis. In patients with thalassemia treated with AQVESME, liver injury with and without jaundice has been observed within the first 6 months of exposure. Obtain liver tests (including ALT, AST, alkaline phosphatase, total bilirubin with fractionation) prior to the initiation of AQVESME, then every 4 weeks for the first 24 weeks, and as clinically indicated thereafter. Interrupt AQVESME if clinically significant increases in liver tests are observed or alanine aminotransferase is >5 times the upper limit of normal (ULN). Complete a comprehensive evaluation to rule out other causes of liver injury when drug-induced liver injury is suspected. Discontinue AQVESME if hepatocellular injury due to AQVESME is suspected.

Symptoms and signs of early liver injury may mimic those of thalassemia. Advise patients to report new or worsening symptoms of loss of appetite, nausea, right-upper-quadrant abdominal pain, vomiting, scleral icterus, jaundice, or dark urine while on AQVESME treatment.

During the double-blind period, 2 of 301 patients (0.66%) with thalassemia treated with AQVESME experienced adverse reactions suggestive of hepatocellular injury. Three additional patients experienced adverse reactions suggestive of hepatocellular injury during the open-label extension periods after switching from placebo to AQVESME. Of these 5 patients, 2 had serious liver injury requiring hospitalization, including 1 patient who developed jaundice (peak bilirubin 32 mg/dL). Another patient developed jaundice (peak bilirubin 4 mg/dL) without requiring hospitalization. These reactions were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5×ULN with or without jaundice. All patients discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation.

AQVESME REMS

AQVESME is available only through a restricted program under a REMS called the AQVESME REMS because of the risk of hepatocellular injury.

ADVERSE REACTIONS

The most common adverse reactions among patients taking AQVESME were headache and insomnia.

DRUG INTERACTIONS

  • Strong CYP3A Inhibitors and Inducers: Avoid concomitant use.
  • Moderate CYP3A Inhibitors: Avoid concomitant use.
  • Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, see full Prescribing Information for recommended dosage for drug interactions with moderate CYP3A inducers.
  • Sensitive CYP3A Substrates, including hormonal contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index.
  • CYP2B6, CYP2C, and UGT1A1 Substrates: Monitor patients for efficacy of the substrates with narrow therapeutic index.
  • P-gp Substrates: Monitor patients for adverse reactions of the substrates with narrow therapeutic index.

HEPATIC IMPAIRMENT

Avoid use of AQVESME in patients with cirrhosis (Child-Pugh Class A, B, or C).

INDICATION

AQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia.

Please see full Prescribing Information for AQVESME, including Boxed Warning.

IMPORTANT SAFETY INFORMATION

BOXED WARNING: HEPATOCELLULAR INJURY

AQVESME can cause serious hepatocellular injury. Measure liver laboratory tests (ALT, AST, alkaline phosphatase and total bilirubin with fractionation) at baseline and every 4 weeks for 24 weeks and then as clinically indicated. Avoid use of AQVESME in patients with cirrhosis. Discontinue AQVESME if hepatic injury is suspected.
Because of the risk of hepatocellular injury, AQVESME is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the AQVESME REMS.

WARNINGS AND PRECAUTIONS

Hepatocellular Injury

AQVESME can cause hepatocellular injury. Avoid use of AQVESME in patients with cirrhosis. In patients with thalassemia treated with AQVESME, liver injury with and without jaundice has been observed within the first 6 months of exposure. Obtain liver tests (including ALT, AST, alkaline phosphatase, total bilirubin with fractionation) prior to the initiation of AQVESME, then every 4 weeks for the first 24 weeks, and as clinically indicated thereafter. Interrupt AQVESME if clinically significant increases in liver tests are observed or alanine aminotransferase is >5 times the upper limit of normal (ULN). Complete a comprehensive evaluation to rule out other causes of liver injury when drug-induced liver injury is suspected. Discontinue AQVESME if hepatocellular injury due to AQVESME is suspected.

Symptoms and signs of early liver injury may mimic those of thalassemia. Advise patients to report new or worsening symptoms of loss of appetite, nausea, right-upper-quadrant abdominal pain, vomiting, scleral icterus, jaundice, or dark urine while on AQVESME treatment.

During the double-blind period, 2 of 301 patients (0.66%) with thalassemia treated with AQVESME experienced adverse reactions suggestive of hepatocellular injury. Three additional patients experienced adverse reactions suggestive of hepatocellular injury during the open-label extension periods after switching from placebo to AQVESME. Of these 5 patients, 2 had serious liver injury requiring hospitalization, including 1 patient who developed jaundice (peak bilirubin 32 mg/dL). Another patient developed jaundice (peak bilirubin 4 mg/dL) without requiring hospitalization. These reactions were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5×ULN with or without jaundice. All patients discontinued treatment with AQVESME, and these reactions improved upon treatment discontinuation.

AQVESME REMS

AQVESME is available only through a restricted program under a REMS called the AQVESME REMS because of the risk of hepatocellular injury.

ADVERSE REACTIONS

The most common adverse reactions among patients taking AQVESME were headache and insomnia.

DRUG INTERACTIONS

  • Strong CYP3A Inhibitors and Inducers: Avoid concomitant use.
  • Moderate CYP3A Inhibitors: Avoid concomitant use.
  • Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, see full Prescribing Information for recommended dosage for drug interactions with moderate CYP3A inducers.
  • Sensitive CYP3A Substrates, including hormonal contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index.
  • CYP2B6, CYP2C, and UGT1A1 Substrates: Monitor patients for efficacy of the substrates with narrow therapeutic index.
  • P-gp Substrates: Monitor patients for adverse reactions of the substrates with narrow therapeutic index.

HEPATIC IMPAIRMENT

Avoid use of AQVESME in patients with cirrhosis (Child-Pugh Class A, B, or C).

INDICATION

AQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia.

Please see full Prescribing Information for AQVESME, including Boxed Warning.