AQVESME

AQVESME is an FDA-registered drug product whose active ingredient is MITAPIVAT. It is labelled by Agios Pharmaceuticals, Inc. taken by oral and marketed as a human prescription drug product. Identifiers on file: NDC 71334-235, application NDA216196, label effective 20260827. It is one of 2 FDA drug label records sharing the active ingredient MITAPIVAT. This page reproduces the FDA-approved label text so you can check what a product is, who makes it and how it is supplied before discussing it with a pharmacist or prescriber.

Across FDA labelling, products containing MITAPIVAT are most often described in connection with: treatment anemia, alpha- beta-thalassemia, usage aqvesme, pyruvate kinase, activator indicated. Wording differs between labels, and the full indications section on this page takes precedence when it is present.

1 INDICATIONS AND USAGE AQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. AQVESME is a pyruvate kinase activator indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. ( 1.1 )

2 DOSAGE AND ADMINISTRATION The tablet should be swallowed whole. Do not split, crush, chew, or dissolve the tablets. ( 2.1 ) Recommended dose is 100 mg orally twice daily with or without food. ( 2.2 ) 2.1 Important Dosage and Administration Information AQVESME is taken with or without food. Swallow tablets whole. Do not split, crush, chew, or dissolve the tablets. If a dose of AQVESME is missed by 4 hours or less, administer the dose as soon as possible. If a dose of AQVESME is missed by more than 4 hours, do not administer a replacement dose, and wait until the next scheduled dose. Subsequently, return to the normal dosing schedule. Monitor for hepatocellular injury during treatment with AQVESME [see Dosage and Administration (2.3) ] . 2.2 Recommended Dosage The recommended dosage for adults with alpha- or beta-thalassemia is AQVESME 100 mg orally twice daily. Treatment with AQVESME is intended to be long-term. Discontinue AQVESME if no benefit in hemolytic anemia has been observed, based on the totality of laboratory results and clinical status of the patient, unless there is another explanation for response failure (e.g., bleeding, surgery, other concomitant illnesses). Interruption or Discontinuation If a patient needs to interrupt or discontinue AQVESME for any reason, a dose taper is not necessary. 2.3 Monitoring for Safety Prior to Initiating Treatment with AQVESME Check liver tests including ALT, AST, alkaline phosphatase, total bilirubin with fractionation, before first AQVESME dose. During Treatment with AQVESME After the first dose, check liver tests including ALT, AST, alkaline phosphatase, total bilirubin with fractionation every 4 weeks for 24 weeks and as clinically indicated thereafter. When Drug-Induced Liver Injury Is Suspected Interrupt AQVESME and complete a comprehensive evaluation to rule out other causes of liver injury. If AQVESME-related liver injury caused new or worsening jaundice or ALT ≥10×baseline, do NOT restart AQVESME. If AQVESME-related liver injury is not ruled out, but peak ALT is <10×baseline without elevation of bilirubin above baseline, and if AQVESME is resumed, reinitiate liver test monitoring every 4 weeks for 24 additional weeks. If AQVESME-related liver injury is ruled out, AQVESME may be restarted at provider discretion. Resume liver test monitoring schedule that existed prior to stopping AQVESME. AQVESME Interruption Due to Non-Liver Causes If AQVESME was stopped for any reason for ≤8 weeks other than suspected AQVESME-related liver injury, resume the liver test monitoring schedule that existed prior to stopping AQVESME. If AQVESME was stopped for more than 8 weeks, restart liver test monitoring every 4 weeks for 24 additional weeks upon resumption of treatment with AQVESME. If treatment is stopped for any duration after 24 weeks of monitoring and treatment, resume monitoring as clinically indicated. 2.4 Recommended Dosage for Drug Interactions Moderate CYP3A Inducers Consider alternative therapies that are not moderate CYP3A inducers during treatment with AQVESME. If there are no alternative therapies, monitor Hb and do not exceed the maximum recommended dose of 100 mg orally twice daily [see Drug Interactions (7.1) and Clinical Pharmacology (12.3) ] .

4 CONTRAINDICATIONS None. None. ( 4 )

6 ADVERSE REACTIONS The following clinically significant adverse reaction is described elsewhere in labeling: Hepatocellular Injury [see Warnings and Precautions (5.1) ]. The most common adverse reactions were headache and insomnia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Agios Pharmaceuticals, Inc. at 1-833-228-8474 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Alpha- and Beta-Thalassemia A total of 301 patients with thalassemia received AQVESME, administered at 100 mg orally twice daily, for up to 59.9 weeks in the ENERGIZE trial (N=129) and the ENERGIZE-T trial (N=172) [see Clinical Studies (14) ] . ENERGIZE Trial Patients with non-transfusion-dependent thalassemia received AQVESME (N=129) or placebo (N=63). The most common adverse reactions (≥5% and at least 5% higher in the AQVESME arm) in patients with non-transfusion-dependent thalassemia were headache and insomnia. ENERGIZE-T Trial Patients with transfusion-dependent thalassemia received AQVESME (N=172) or placebo (N=85). The most common adverse reactions (≥5% and at least 5% higher in the AQVESME arm) in patients with transfusion-dependent thalassemia were headache and insomnia. Serious adverse reactions occurred in 1.3% of patients with thalassemia treated with AQVESME, including supraventricular arrhythmia and supraventricular tachycardia. Permanent discontinuations of AQVESME due to an adverse reaction occurred in 1.3% of patients and included elevated hepatic transaminases and insomnia. Table 1 summarizes the adverse reactions in the ENERGIZE and the ENERGIZE-T trials, individually and combined. Table 1: Adverse Reactions a in Patients with Alpha- and Beta-Thalassemia Receiving AQVESME ENERGIZE (Non-transfusion-dependent) ENERGIZE-T (Transfusion-dependent) Total Adverse Reactions AQVESME (N=129) n (%) Placebo (N=63) n (%) AQVESME (N=172) n (%) Placebo (N=85) n (%) AQVESME (N=301) n (%) Placebo (N=148) n (%) Headache 29 (22.5) 6 (9.5) 46 (26.7) 10 (11.8) 75 (24.9) 16 (10.8) Insomnia b 35 (27.1) 5 (7.9) 38 (22.1) 8 (9.4) 73 (24.3) 13 (8.8) a Included adverse reactions that occurred in at least 5% of patients in the AQVESME arm and at least 5% higher than the placebo arm. b Term includes initial insomnia, middle insomnia, and terminal insomnia. Variations in Reproductive Hormones Increases in serum testosterone (T) concentrations and decreases in serum estradiol (E2) concentrations were observed in men receiving AQVESME (Table 2). These changes in hormones were maintained during treatment with AQVESME. In 3 male patients who discontinued AQVESME and in whom reproductive hormone data were available following discontinuation of AQVESME, the hormone changes were reversible. In female patients, sex hormone analysis was limited due to physiologic variations in hormones during the menstrual cycle and the use of hormonal contraceptives. Table 2: Abnormalities in Reproductive Hormones in Men with Thalassemia Receiving AQVESME ENERGIZE (Non-transfusion-dependent) ENERGIZE-T (Transfusion-dependent) Parameter AQVESME (46 males) Placebo (25 males) AQVESME (64 males) Placebo (31 males) Reproductive hormone analyses Testosterone (T) Serum T concentration (mean) Baseline Change from baseline 613 ng/dL 228 ng/dL 505 ng/dL -2.8 ng/dL 625 ng/dL 108 ng/dL 666 ng/dL 66 ng/dL Serum T increased a Baseline Change from baseline 2.2% 18.6% 4.3% 0% 3.3% 13.5% 10% 6.9% Estradiol (E2) Serum E2 concentration (mean) Baseline Change from baseline 29.6 pg/mL -8.7 pg/mL 27.3 pg/mL 0 pg/mL 26.4 pg/mL -5.0 pg/mL 28.9 pg/mL 1.6 pg/mL Serum E2 decreased b Baseline Change from baseline 0% 2.5% 9.5% 0% 10.2% 8.5% 6.9% 3.6% a Percentage of subjects with serum T concentration above the upper limit of normal (greater than 1050 ng/dL) at baseline and percentage of subjects with serum T increases from baseline to above the upper limit of normal where baseline was within normal limits. b Percentage of subjects with serum E2 concentration below the lower limit of normal at baseline and percentage of subjects with serum E2 decreases from baseline to below the lower limit of normal where baseline was within normal limits. Note: Results from the ENERGIZE-T study do not include data from patients who received concomitant testosterone replacement therapies.

FDA lists 2 drug label records with MITAPIVAT as the active ingredient. The most common dosage forms and the manufacturers with the most records are summarised below; strengths, pack sizes and inactive ingredients can still differ between apparently similar products.

What is AQVESME used for?
1 INDICATIONS AND USAGE AQVESME is indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. AQVESME is a pyruvate kinase activator indicated for the treatment of anemia in adults with alpha- or beta-thalassemia. ( 1.1 )

Who makes AQVESME?
Agios Pharmaceuticals, Inc. is the labeler of record for AQVESME. 2 FDA label records share the active ingredient MITAPIVAT, so generic and branded versions may come from different companies; the manufacturer named on your pack is the one that matters for any enquiry.

What form does AQVESME come in?
administered by oral. Strengths and pack sizes vary by label, so compare the NDC on your pack with the identifier listed on this page.

Is AQVESME prescription or over-the-counter?
The FDA label for this product is recorded as human prescription drug. Marketing status can differ between strengths and countries, so check the dispensing rules where you live.

Are there other products containing MITAPIVAT?
Yes. 2 FDA drug label records list MITAPIVAT as the active ingredient. Products with the same active ingredient can usually be substituted only when a prescriber or pharmacist confirms it is appropriate.

Has AQVESME been recalled?
No FDA recall record in our database matches this product name or manufacturer. Recall data is updated periodically, so a very recent notice may not appear here yet.

Where does this information come from?
All content is reproduced from FDA Structured Product Labelling data via openFDA. It is provided for identification and reference only and is not medical advice.