TheraRadar
Data updated: May 26, 2026

AQVESME (mitapivat sulfate)

Pyruvate Kinase Activators
Rare Disease Approved 2022-02-17

Aqvesme treats anemia in adults who have alpha- or beta-thalassemia. This medication helps patients by addressing the underlying issues that cause red blood cells to break down or develop improperly. It is used to improve red blood cell health and manage the chronic low blood counts associated with these genetic conditions.

Source: FDA Label • AGIOS PHARMS INC • Pyruvate Kinase Activator

How AQVESME Works

This medication works by binding to the pyruvate kinase enzyme to increase its activity and improve energy balance within red blood cells. By boosting this enzyme, the drug helps reduce oxidative stress and cell destruction while improving the lifespan and production of healthy red blood cells.

2
Indications
--
Phase 3 Trials
1
Priority Reviews
4
Years on Market

Details

Status
Prescription
First Approved
2022-02-17
Patent Cliff
2041

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Routes
ORAL
Dosage Forms
TABLET

Companies

Active Ingredient: MITAPIVAT SULFATE

AQVESME Approval History

2023
2024
2025
2026
Original
New Indication
New Form
Label Update
3 FDA actions from 2022 to 2025 · 1 indication expansions
Dec 2025 SUPPL
Efficacy
Jan 2025 SUPPL
Label · Labeling
Feb 2022 ORIGINAL Priority
New Drug · Type 1 - New Molecular Entity

What AQVESME Treats

2 indications

AQVESME is approved for 2 conditions since its original approval in 2022. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Anemia
  • Thalassemia
Source: FDA Label

AQVESME 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 [see Warnings and Precautions ( 5.1 )] . Because of the risk of hepatocellular injury, AQVESME is available only through a restricted program under ...

AQVESME Competitive Set

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Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.

Drugs Similar to AQVESME

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DEFERASIROX
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PHARMOBEDIENT
Shared indications:
Thalassemia
DEFERIPRONE
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Hikma
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EPOGEN/PROCRIT
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Amgen
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Clinical Trial Registry

2 trials
Trial Sponsor ID Phase Status Title
NCT05935202 SATISFY SATISFY Ph 2 not yet recruiting Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies
NCT03991312 AG348-C-012 Ph 1 completed Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348) in Healthy Adult Participants
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Active Pipeline

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Key Completed Trials

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Trial Timeline

Full development history with FDA approval milestones

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

AQVESME FDA Label Details

Indications & Usage

FDA Label (PDF)

AQVESME is indicated for the treatment of Anemia; Thalassemia.

⚠️ BOXED WARNING

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 c...

View full patent landscape →
9 OB patents · 5 families · 273 international docs across 48 countries

AQVESME Patents & Exclusivity

Latest Patent: Jul 2041
Exclusivity: Dec 2032

Patents (9 active)

US11878049 Expires Jul 31, 2041
US11254652 Expires Nov 21, 2038
US11234976 Expires Oct 11, 2038
USRE49582 Expires Apr 11, 2035
US11793806 Expires Apr 12, 2033
US9193701 Expires Oct 26, 2032
US10632114 Expires May 3, 2032
US9682080 Expires May 3, 2032
US9980961 Expires May 3, 2032

Exclusivity

NCE Until Feb 2027
ODE-392 Until Feb 2029
NCE Until Feb 2027
ODE-392 Until Feb 2029
NCE Until Feb 2027
ODE-392 Until Feb 2029
NCE Until Feb 2027
NP Until Dec 2028
ODE-522 Until Dec 2032
Source: FDA Orange Book

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Patent Timeline

  • Cliff: 2041
  • 33 active patents

Trial Analysis

  • Clinical trial tracking
  • Development stage analysis

Competitive Landscape

  • 15 similar drugs
  • Same target/indication analysis
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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.