In NTDT patients, AQVESME demonstrated significant improvement in Hb levels and reduction in fatigue vs placebo1

A higher percentage of patients on AQVESME achieved a clinically meaningful increase of ≥1.0 g/dL in Hb levels from baseline vs placebo1,2*

A waterfall plot showing change from baseline in average Hb concentration for AQVESME vs placebo from weeks 12 through 24

1.6 g/dL average Hb increase among AQVESME-treated patients who met the primary endpoint1‡

Hb=hemoglobin; HbH=hemoglobin H; NTDT=non–transfusion-dependent thalassemia.

*All randomized patients.1

†P value is 2-sided, and all results are statistically significant (95% CI). Difference adjusted for randomization stratification factors, including Hb concentrations (≤9.0 g/dL vs 9.1-10.0 g/dL) and genotype (α-thalassemia/HbH disease vs β-thalassemia). Two-sided P value was based on the Mantel-Haenszel stratum weighted method, adjusting for these randomization stratification factors.1

‡Among the 55 patients in the AQVESME treatment arm who met the primary endpoint, defined as a ≥1 g/dL increase from baseline in average Hb from week 12 through week 24, the average Hb increase over weeks 12-24 was 1.6 g/dL.1

Fatigue reduction

AQVESME demonstrated a significant improvement from baseline in FACIT-Fatigue score vs placebo1

Bar chart showing improvement from baseline in average FACIT-Fatigue score with AQVESME from week 12 through 24 vs placebo

What is FACIT-Fatigue?

A self-reported, validated questionnaire that is used to measure fatigue and tiredness over the previous week on a scale of 0 to 52.3

Higher scores indicate less fatigue.3

The US general population mean score on the FACIT-Fatigue scale is 44.4

FACIT-Fatigue=Functional Assessment of Chronic Illness Therapy–Fatigue Scale.

Contact an Agios rep to learn more about AQVESME efficacy in NTDT

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Explore the impact of AQVESME on transfusion burden in TDT patients

EXPLORE TDT RESULTS

TDT=transfusion-dependent thalassemia.

References: 1. AQVESME. Prescribing Information. Agios Pharmaceuticals, Inc.; 2025. 2. Taher AT et al. Guidelines for the Management of Non-Transfusion-Dependent β-Thalassaemia. 3rd ed. Thalassaemia International Federation; 2023. 3. Acaster S et al. Health Qual Life Outcomes. 2015;13:60. doi:10.1186/s12955-015-0257-x 4. Cella D et al. Cancer. 2002;94(2):528-538. doi:10.1002/cncr.10245

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.