Mitapivat

Key takeaways

Mitapivat belongs to chemistry; place it in that map before memorising details.
Learn the definition first, then one example that makes the definition concrete.
Connect Mitapivat to a quantity you can measure, compute or draw — that is where exam questions come from.
Reproduce the core statement of Mitapivat from memory before moving on to harder problems.

Reference excerpt

Mitapivat, sold under the brand name Pyrukynd among others, is a medication used to treat different types of anemia. It is taken as the sulfate hydrate salt by mouth. Mitapivat is a pyruvate kinase activator. The most common side effects include decreases in estrone and estradiol (types of the estrogen hormone) in men, increased urate (a type of salt in the body), back pain, and joint stiffness. Mitapivat was approved for medical use in the United States in February 2022, and in the European Union in November 2022. The US Food and Drug Administration considers it to be a first-in-class medication.

Medical uses Mitapivat is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. In December 2025, mitapivat (brand name Aqvesme) was approved in the US for the treatement of anemia in adults with alpha- or beta-thalassemia. Mitapivat has also been evaluated for the treatment of sickle cell disease, with a promising phase 2 trial.

Pharmacology

Mechanism of action Mitapivat binds to and activates pyruvate kinase, thereby enhancing glycolytic pathway activity, improving adenosine triphosphate (ATP) levels and reducing 2,3-diphosphoglycerate (2,3-DPG) levels. Mutations in pyruvate kinase cause deficiency in pyruvate kinase which prevents adequate red blood cell (RBC) glycolysis, leading to a buildup of the upstream glycolytic intermediate 2,3-DPG and deficiency in the pyruvate kinase product ATP.

History The US Food and Drug Administration approved mitapivat based on evidence from two clinical trials of 107 participants with pyruvate kinase deficiency. Trial 1 (NCT03548220) of 80 adults with pyruvate kinase deficiency who did not receive regular blood transfusions and trial 2 (NCT03559699) of 27 adults with pyruvate kinase deficiency who received regular blood transfusions. In trial 1, participants were randomly assigned to receive either mitapivat or a matched placebo tablet for an average duration of about 24 weeks. Neither the participants nor the healthcare providers knew which treatment was being given during the trial. Trial 1 was conducted at 36 sites in the following countries: Brazil, Canada, Denmark, France, Germany, Italy, Japan, Republic of Korea, Netherlands, Spain, Switzerland, Turkey, United Kingdom, and the United States. In Trial 2, all participants received mitapivat for an average duration of about 40 weeks. Trial 2 was conducted at 17 sites in the following countries: Canada, Denmark, France, Ireland, Italy, Netherlands, Thailand, United Kingdom, and the United States.

Society and culture

Legal status In September 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Pyrukynd, intended for the treatment of an inherited condition called pyruvate kinase deficiency. The applicant for this medicinal product is Agios Netherlands B.V. Mitapivat was approved for medical use in the European Union in November 2022.

Names Mitapivat is the international nonproprietary name (INN). Mitapivat is sold under the brand names Pyrukynd and Aqvesme.

References

Further reading Kung C, Hixon J, Kosinski PA, Cianchetta G, Histen G, Chen Y, et al. (September 2017). "AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency". Blood. 130 (11): 1347–1356. doi:10.1182/blood-2016-11-753525. PMC 5609468. PMID 28760888. Rab MA, Van Oirschot BA, Kosinski PA, Hixon J, Johnson K, Chubukov V, et al. (January 2021). "AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes". Haematologica. 106 (1): 238–249. doi:10.3324/haematol.2019.238865. PMC 7776327. PMID 31974203.

External links Clinical trial number NCT03548220 for "A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)" at ClinicalTrials.gov Clinical trial number NCT03559699 for "A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)" at ClinicalTrials.gov Clinical trial number NCT04770779 for "A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T)" at ClinicalTrials.gov Clinical trial number NCT04770753 for "A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)" at ClinicalTrials.gov

Illustrations

Worked examples

Example 1 — a first encounter with Mitapivat

Start with the simplest possible case. Write down what Mitapivat claims or describes in one sentence, then invent the smallest concrete situation in which that sentence is true. In chemistry, the smallest case is usually a single object, a single equation or a single measurement. Check that every symbol or term in your sentence has a meaning in that case.

Example 2 — changing one variable

Take the situation from Example 1 and change exactly one quantity: double it, halve it, or set it to zero. Predict what should happen to Mitapivat before you calculate. Comparing your prediction with the result is the fastest way to find out whether you understand the idea or only the words.

Example 3 — an exam-style question

Typical questions about Mitapivat ask you to (a) state it precisely, (b) apply it to given data, and (c) explain a limitation. Practise writing all three answers in under five minutes; the third part is what separates a full-mark answer from an average one.

Applications of Mitapivat

In research: Mitapivat appears in chemistry research whenever the underlying quantities have to be modelled precisely. Papers usually cite it as a starting assumption and then explore where it breaks down.
In technology and industry: Engineering practice reuses Mitapivat in design rules, simulations and safety margins. Knowing the idea lets you read a specification sheet and understand why the numbers look the way they do.
In the classroom: Mitapivat is common in secondary-school and first-year university syllabi. It links to neighbouring topics Anilines, CYP3A4 inducers, Cyclopropyl compounds, so understanding it makes those chapters shorter.
In everyday life: Look for Mitapivat outside the textbook — in sport, cooking, traffic, electronics or the sky above you. An example you found yourself is remembered far longer than one you were given.

How to study Mitapivat in 20 minutes

Read the reference excerpt below once, without taking notes.
Close the page and write down what Mitapivat means in your own words.
Compare your version with the excerpt and mark what you missed.
Work through the three examples above with pen and paper.
Explain Mitapivat out loud to somebody else — or to Teacher Smith in the lgStudy chat.

Frequently asked questions

What is Mitapivat in simple terms?

Mitapivat, sold under the brand name Pyrukynd among others, is a medication used to treat different types of anemia. It is taken as the sulfate hydrate salt by mouth.

Why does Mitapivat matter?

Because it connects several chemistry ideas at once: it gives you a definition you can apply, a quantity you can calculate, and a way to check whether a result is plausible.

How should I study Mitapivat?

Read the excerpt, restate it from memory, then work through the examples and applications listed on this page. The five-step study plan above takes about twenty minutes.

What does this page cover?

It gives you a compact reference excerpt plus original lgStudy explanations, examples, applications and study material on Mitapivat.