Mitapivat
Pronunciation: (mih-TAH-pih-vat).
- Generic name
- Mitapivat
- Brand names
- Pyrukynd, Pyruvact, Mitakynd, Mitapiva
- Drug class
- Endocrine & Metabolic Disorders
Availability and supply rules vary by country
Trade names by country: United States
Australia: · Full directory
What Mitapivat is and how it works
Uses, formulations, monitoring, and supply rules can differ by patient, product, and country. Follow the exact local product information and professional advice.
Mechanism of action: how Mitapivat works
Mitapivat is an oral, allosteric activator of the pyruvate kinase (PK) enzyme in red blood cells. By increasing PK activity, mitapivat enhances glycolytic flux, leading to improved adenosine triphosphate (ATP) production and red cell survival. This reduces hemolysis and increases hemoglobin levels in patients with PK deficiency.
What Mitapivat is used for
Pyruvate kinase deficiency
Pyruvate kinase deficiency (2)
Before taking Mitapivat
Do not take / not appropriate for
- Contraindicated in patients with known hypersensitivity to mitapivat or any excipients. (Contraindication) (2)
Tell your clinician about
(2)How to take Mitapivat
- General Instructions: Take Mitapivat exactly as prescribed, usually twice daily with or without food. Swallow the tablet whole with water.. Adult Dosing: The starting dose is 5 mg orally twice daily. Based on hemoglobin response and tolerability, the prescriber may titrate the dose upward — to 20 mg twice daily, and then to a maximum of 50 mg twice daily — with dose increases spaced at least 4 weeks apart. Some patients reach an adequate response at 5 mg or 20 mg twice daily and do not need further increases.. Pediatric Dosing: Safety and efficacy in children under 18 years have not been established. Use in pediatric patients is not recommended.. Administration: Take at the same times each day. Do not crush, chew, or split the tablets.. When To Stop: Seek medical advice before stopping Mitapivat, even if you feel well — stopping abruptly has been linked to a flare of red-blood-cell breakdown (hemolysis) and worsening anemia; let the clinician you see guide a safe tapering schedule if treatment needs to end. Report jaundice, dark urine, dizziness, unusual fatigue, or shortness of breath promptly.. Storage: Store at room temperature (20–25°C/68–77°F), away from moisture and heat, in the original carton. Keep out of reach of children.
- This page does not provide a dose calculator. Dosing must follow the prescriber, pharmacist, or product label.
Procedures and treatment changes
Tell the treating team before surgery, procedures, or any medicine change. Do not alter treatment without the responsible clinician. (2)
Missed dose
If you miss a dose, take it as soon as you remember. If it is almost time for your next dose, skip the missed dose and resume your regular schedule. Do not take two doses at once.
Overdose
If you or someone else takes too much Mitapivat, seek emergency medical attention or contact a poison control center immediately. Symptoms of overdose may include severe headache, confusion, or worsening side effects. (2)
Side effects
Side effects and their urgency vary; report severe, persistent, or concerning symptoms to a healthcare professional.
Nausea
Incidence: Reported in the clinician-authored database; frequency not established
Diarrhea
Incidence: Reported in the clinician-authored database; frequency not established
Headache
Incidence: Reported in the clinician-authored database; frequency not established
Insomnia
Incidence: Reported in the clinician-authored database; frequency not established
Arthralgia
Incidence: Reported in the clinician-authored database; frequency not established
Anemia
Incidence: Reported in the clinician-authored database; frequency not established
Hemolysis
Incidence: Reported in the clinician-authored database; frequency not established
Serious warnings
Acute hemolysis (red blood cell breakdown) with worsening anemia has been reported after abruptly stopping or interrupting mitapivat — serious
Symptoms: Acute hemolysis (red blood cell breakdown) with worsening anemia has been reported after abruptly stopping or interrupting mitapivat. Any change to treatment, including stopping it, should be directed by the prescriber using a gradual taper. Seek prompt medical advice for jaundice, dark urine, dizziness, unusual fatigue, or shortness of breath.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2)
Monitor liver function regularly during treatment — serious
Symptoms: Monitor liver function regularly during treatment. Use caution in patients with renal impairment; avoid use in moderate-to-severe hepatic impairment. Not recommended during pregnancy or breastfeeding unless clearly needed. May interact with other medications metabolized by CYP3A. Report any signs of anemia, jaundice, or allergic reaction promptly.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2)
The FDA label lists no formal contraindications for mitapivat — serious
Symptoms: The FDA label lists no formal contraindications for mitapivat. Avoid use if you have had a serious allergic reaction to mitapivat or its other ingredients. Renal and hepatic impairment require special caution — avoid use in moderate-to-severe hepatic impairment specifically. Monitor for potential drug interactions, especially with CYP3A modulators.
Action: Follow the urgency stated and seek prompt or emergency care when indicated. (2)
Interactions
| Medicine or test | Effect | Action |
|---|---|---|
| Recorded interactions | Mitapivat may interact with drugs that induce or inhibit CYP3A enzymes, such as certain antifungals, antibiotics, and anticonvulsants. These interactions can alter mitapivat blood levels and effectiveness. Always inform your healthcare provider about all medications and supplements you are taking. | |
| Strong CYP3A inhibitors (e.g., itraconazole, ketoconazole) | Combining these with mitapivat is generally avoided, as they can raise mitapivat levels substantially (about 3.5-5-fold in studies), increasing the risk of side effects. | (2) |
| Moderate CYP3A inhibitors (e.g., fluconazole) | If used together, the prescriber generally will not increase the mitapivat dose beyond 20 mg twice daily. | (2) |
| Strong CYP3A inducers (e.g., rifampin) | Combining these with mitapivat is generally avoided, as they can sharply lower mitapivat levels (roughly 90% or more), risking loss of effectiveness. | (2) |
| Moderate CYP3A inducers (e.g., efavirenz) | The prescriber will generally consider an alternative; if the combination is necessary, they may need to titrate mitapivat to a higher-than-usual dose (up to 100 mg twice daily) to maintain effect. | (2) |
Pregnancy, breastfeeding, kidney/liver function, age
Pregnancy
Available clinical-trial data are insufficient to evaluate whether mitapivat raises the risk of birth defects. Animal studies at doses up to 13 times the maximum recommended human dose showed no harm to the fetus. Untreated PK deficiency itself carries risks in pregnancy (such as acute hemolysis and preeclampsia), so the decision to continue or start mitapivat during pregnancy should be made together with the prescribing team, weighing these factors. (2)
Breastfeeding
There is no information on whether mitapivat or its breakdown products pass into human or animal breast milk. Discuss the risks and benefits of breastfeeding while taking mitapivat with your healthcare provider. (2)
Children
Frequency: Not applicable. Weight Based: Not recommended for pediatric use. Max Daily Dose: Not applicable. Max Single Dose: Not applicable. Age Restrictions: Safety not established in children (2)
Renal_impairment
Hepatic impairment: mitapivat should generally be AVOIDED in moderate or severe hepatic impairment (higher drug exposure seen in moderate impairment; no data in severe impairment). Renal impairment: no dose adjustment needed for eGFR 60 mL/min/1.73m^2 or above; data are limited for eGFR 30 to under 60, and there is no data for eGFR below 30, so extra caution and prescriber judgment are needed in more significant kidney impairment. (2)
Hepatic_impairment
Hepatic impairment: mitapivat should generally be AVOIDED in moderate or severe hepatic impairment (higher drug exposure seen in moderate impairment; no data in severe impairment). Renal impairment: no dose adjustment needed for eGFR 60 mL/min/1.73m^2 or above; data are limited for eGFR 30 to under 60, and there is no data for eGFR below 30, so extra caution and prescriber judgment are needed in more significant kidney impairment. (2)
Monitoring
(2)Storage
Store mitapivat at controlled room temperature, 20-25C (68-77F), with brief excursions to 15-30C permitted, in the original carton until use, away from moisture and heat, and out of reach of children.
Professional information
Clinician-facing context only. This section does not replace current product labeling.
General
Indication: Pyruvate kinase deficiency
Dose summary: Start at 5 mg orally twice daily; titrate to 20 mg twice daily and then to a maximum of 50 mg twice daily, with increases at least 4 weeks apart, assessing hemoglobin/transfusion need before each step. Baseline and monthly (first 6 months) liver enzyme monitoring; interrupt if ALT >5x ULN. Discontinue after 24 weeks if no meaningful hematologic benefit.
Renal context: No dose adjustment needed for eGFR >=60 mL/min/1.73m^2. Limited data for eGFR 30-<60 mL/min/1.73m^2; no data for eGFR <30 mL/min/1.73m^2. (2)
Frequently asked questions
What is Mitapivat used for?
Mitapivat is used to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare inherited blood disorder.
How does Mitapivat work?
Mitapivat activates the pyruvate kinase enzyme in red blood cells, improving their energy production and survival, which helps reduce anemia.
What are the most common side effects?
Common side effects include headache, nausea, fatigue, joint pain, and mild changes in liver enzymes.
Can I take Mitapivat if I am pregnant or breastfeeding?
Mitapivat is not recommended during pregnancy or breastfeeding unless clearly needed. Discuss risks and benefits with your doctor.
What should I do if I miss a dose?
Take the missed dose as soon as you remember, unless it is almost time for your next dose. Do not take an extra dose.
Are there any foods or drugs I should avoid?
Avoid grapefruit and grapefruit juice, and inform your doctor about all medications, especially those affecting CYP3A enzymes.
How often will I need blood tests while taking Mitapivat?
Regular blood tests are required to monitor your hemoglobin levels, liver function, and overall response to treatment.
What happens if I take too much Mitapivat?
Taking too much Mitapivat can lead to an exacerbation of side effects. While specific overdose symptoms are not fully established, it is crucial to seek immediate medical attention or contact a poison control center if an overdose is suspected. Do not attempt to self-treat an overdose. Prompt emergency care is essential to manage potential adverse reactions.
Can I drink alcohol while taking Mitapivat?
The interaction between Mitapivat and alcohol has not been extensively studied. However, as a pyruvate kinase activator, Mitapivat affects metabolic pathways. Alcohol can also impact liver function and metabolism, potentially leading to additive effects or increased risk of side effects. It is generally advisable to consult your healthcare provider regarding alcohol consumption while on Mitapivat.
Is Mitapivat safe during pregnancy?
Mitapivat's safety during pregnancy has not been definitively established. It is categorized as 'Consult doctor' regarding pregnancy. Therefore, if you are pregnant, planning to become pregnant, or breastfeeding, it is imperative to discuss the potential risks and benefits of Mitapivat with your healthcare provider. They can provide personalized advice based on your individual circumstances.
What is the mechanism of action of Mitapivat?
Mitapivat is an oral, allosteric activator of the pyruvate kinase (PK) enzyme in red blood cells. By increasing PK activity, mitapivat enhances glycolytic flux, leading to improved adenosine triphosphate (ATP) production and red cell survival. This reduces hemolysis and increases hemoglobin levels in patients with PK deficiency.
What is the generic name of Mitapivat?
The generic name is Mitapivat. It is sold under brand names including Pyrukynd, Pyruvact, Mitakynd, Mitapiva.
What class of drug is Mitapivat?
Mitapivat is classed as: Endocrine & Metabolic Disorders.
References
- Mitapivat medicine recordMedGuideGlobal Clinician‑Authored Medicine Record · International (internal database) · accessed 2026-08-06 · source 1
- PYRUKYND (mitapivat) tablets – full prescribing informationU.S. Food and Drug Administration · United States · accessed 2026-08-06 · source 2
- PYRUKYND (MITAPIVAT) TABLET, FILM COATED PYRUKYND (MITAPIVAT) KIT [AGIOS PHARMACEUTICALS, INC.] — US prescribing information (DailyMed SPL)U.S. National Library of Medicine (DailyMed) · US · accessed 2026-08-14 · source 3
- Mitapivat (Hemolytic Anemia) — patient drug information (MedlinePlus)U.S. National Library of Medicine (MedlinePlus) · US · accessed 2026-08-14 · source 4
- AQVESME — FDA approval record (Drugs@FDA NDA216196)U.S. Food and Drug Administration · US · accessed 2026-08-14 · source 5
- Mitapivat — RxNorm normalized drug concept (RXCUI 2594468)U.S. National Library of Medicine (RxNorm) · US · accessed 2026-08-14 · source 6
Brand names by country
Single-ingredient Mitapivat names are shown separately by country. Product availability, strengths, and supply rules can change; combination products are not included here.
Priority countries
United States
- PYRUKYND5 mg, 20 mg, 50 mg · agios pharmaceuticals, inc.
- AQVESME100 mg · agios pharmaceuticals, inc.