Mitapivat
Mitapivat is a pyruvate kinase activator taken by mouth to treat anemia in adults, either hemolytic anemia from pyruvate kinase deficiency or anemia from alpha- or beta-thalassemia, depending on the brand.
🧬 Where this information comes from Click to expand
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 2 clinical label sets within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
Mitapivat is a newer specialist medicine, on the U.S. market since about 2022, that boosts pyruvate kinase, an enzyme red blood cells need for energy, to treat anemia in adults with PK deficiency (Pyrukynd) or alpha- or beta-thalassemia (Aqvesme). It is generally well tolerated, and headache and trouble sleeping are the side effects people notice most. Aqvesme carries a boxed warning for serious liver injury, so it comes only through a restricted program (REMS), with liver blood tests every 4 weeks for the first 24 weeks. With Pyrukynd, do not stop on your own, since stopping suddenly can trigger a burst of red blood cell breakdown.
Clinical pearls
- Swallow the tablets whole and never split, crush, chew or dissolve them, though food makes no difference.
- On Aqvesme, report yellow skin or eyes, dark urine, nausea or upper right belly pain, which can mimic thalassemia symptoms.
- Aqvesme weakens hormonal birth control except a levonorgestrel IUD, so add non-hormonal backup during use and for 28 days after stopping.
- Many drugs that slow or speed liver breakdown (CYP3A inhibitors or inducers) shift mitapivat levels, so have every new prescription checked.
⚠️ FDA Boxed Warning — the FDA’s strongest safety alert ▾
Aqvesme can cause serious liver injury. This is the most important risk of the brand.
Your doctor will check liver blood tests before you start, every 4 weeks for the first 24 weeks, and later as needed. People with cirrhosis should avoid Aqvesme. Stop and call your doctor if you suspect liver injury, such as yellow skin or eyes, dark urine, nausea, vomiting, loss of appetite or upper right belly pain.
Because of this risk, Aqvesme is available only through a restricted program called the Aqvesme REMS. Prescribers, pharmacies and patients must all enroll.
Patient information guide A plain-language walkthrough of Mitapivat, written from its FDA label.
What Mitapivat is used for
▾
Mitapivat is used for inherited anemias in adults, depending on the brand.
- Pyrukynd (oral tablet): hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
- Aqvesme (oral tablet): anemia in adults with alpha- or beta-thalassemia.
How Mitapivat works
▾
Mitapivat is a pyruvate kinase activator. It attaches to the pyruvate kinase enzyme and increases its activity. In PK deficiency, the red blood cell form of this enzyme is mutated, which lowers cell energy (ATP), shortens red cell lifespan and causes ongoing hemolysis.
In thalassemia, imbalanced hemoglobin production causes stress on red cells and poor red cell production. In lab models of beta-thalassemia, boosting the enzyme improved red cell energy and lifespan and reduced hemolysis.
Mitapivat dosage
▾
Both brands are film-coated tablets taken by mouth twice daily, with or without food. Swallow them whole.
- Pyrukynd: started at a low dose and raised step by step every 4 weeks based on hemoglobin and transfusion needs. It is stopped if no benefit by 24 weeks. Tapering is preferred over stopping suddenly.
- Aqvesme: one recommended dose, intended for long-term use. It is stopped if there is no benefit. No taper is needed.
- Missed dose (both): if 4 hours late or less, take it as soon as possible. If more than 4 hours late, skip it and take the next dose on schedule.
Follow your prescriber and the label for your exact dose.
Dosing details from the FDA-approved label
From the Mitapivat prescribing information (“Dosage and Administration”). Doses are set by your prescriber for your product, condition, kidney function and age — never change a dose on your own.
- Pyrukynd (pyruvate kinase deficiency): starting dosage and titration
- Starting dosage 5 mg orally twice daily with or without food, swallowed whole
- Titrate every 4 weeks from 5 mg twice daily to 20 mg twice daily, then to 50 mg twice daily
- Weeks 5-8: if Hb is below normal range or transfusion within the last 8 weeks, increase to 20 mg twice daily for 4 weeks
- If Hb is within normal range and no transfusion within the last 8
- Maximum: 50 mg twice daily (up to 100 mg twice daily with moderate CYP3A inducers if no alternatives; do not titrate beyond 20 mg twice daily with a moderate CYP3A inhibitor)
- Discontinue if no benefit by 24 weeks. Do not split, crush, chew, or dissolve tablets. Missed dose by 4 hours or less: take as soon as possible; more than 4 hours: skip it.
- Pyrukynd: dose taper for interruption or discontinuation
- 5 mg twice daily: 5 mg once daily Day 1-7, then discontinue Day 8. 20 mg twice daily: 20 mg once daily Day 1-7, 5 mg once daily Day 8-14, discontinue Day 15. 50 mg twice daily: 50 mg once daily Day 1-7, 20 mg once daily Day 8-14, discontinue Day 15
- Avoid abrupt discontinuation to reduce risk of acute hemolysis; for adverse reactions or Hb above normal, dose may be reduced to 20 mg or 5 mg twice daily.
- Aqvesme: adults with alpha- or beta-thalassemia
- 100 mg orally twice daily with or without food, swallowed whole
- Treatment is intended to be long-term
- No dose taper needed for interruption or discontinuation. Missed dose by 4 hours or less: take as soon as possible; more than 4 hours: skip it. Check liver tests before first dose and every 4 weeks for 24 weeks.
Read the label’s full dosing text
2 DOSAGE AND ADMINISTRATION Starting dosage: 5 mg orally twice daily with or without food. ( 2.1 ) See Full Prescribing Information for dose titration and taper schedule. ( 2.1 , 2.3 ) The tablet should be swallowed whole. ( 2.1 ) 2.1 Recommended Dose PYRUKYND is taken with or without food and swallowed whole. Do not split, crush, chew, or dissolve the tablets. The starting dosage for PYRUKYND is 5 mg orally twice daily. To gradually increase hemoglobin (Hb), titrate PYRUKYND from 5 mg twice daily to 20 mg twice daily, and then to the maximum recommended dose of 50 mg twice daily, with these dose increases occurring every 4 weeks (see Table 1). Assess Hb and transfusion requirement before increasing to the next dose level, as some patients may reach and maintain normal Hb at 5 mg twice daily or 20 mg twice daily. Discontinue PYRUKYND if no benefit has been observed by 24 weeks, based on the hemoglobin and hemolysis laboratory results and transfusion requirements. Table 1: Dose Titration Schedule Duration Dosage Week 1 through Week 4 5 mg twice daily Week 5 through Week 8 If Hb is below normal range or patient has required a transfusion within the last 8 weeks: Increase to 20 mg twice daily and maintain for 4 weeks. If Hb is within normal range and patient has not required a transfusion within the last 8 weeks: Maintain 5 mg twice daily. Week 9 through Week 12 If Hb is below normal range or patient has required a transfusion within the last 8 weeks: Increase to 50 mg twice daily and maintain thereafter. If Hb is within normal range and patient has not required a transfusion within the last 8 weeks: Maintain current dose (5 mg twice daily or 20 mg twice daily). Maintenance If Hb decreases, consider up-titration to the maximum of 50 mg twice daily as per the above schedule. 2.2 Missed Dose If a dose of PYRUKYND is missed by 4 hours or less, administer the dose as soon as possible. If a dose of PYRUKYND 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. 2.3 Interruption or Discontinuation To reduce the risk of acute hemolysis, avoid abrupt interruption or abrupt discontinuation of PYRUKYND when possible [see Warnings and Precautions (5.1) ] . Taper the dose to gradually discontinue the medication (see Table 2).
Excerpted — the section continues on the label. Read the full Dosage and Administration section on DailyMed →
Mitapivat side effects
▾
The most common side effects reported with Aqvesme in studies were:
Common side effects
- Headache
- Trouble sleeping (insomnia)
- Changes in reproductive hormones in men (higher testosterone, lower estradiol), seen with Aqvesme
- Higher testosterone and lower estradiol in men, which reversed after stopping in the few men checked
When to get medical help
- Call your doctor right away for yellow skin or eyes (jaundice), or dark urine, while taking Aqvesme.
- Report new or worsening loss of appetite, nausea, vomiting or pain in the upper right belly. These can signal liver injury.
- Tell your doctor about any irregular or fast heartbeat. Serious heart rhythm problems (supraventricular arrhythmia and tachycardia) were reported with Aqvesme.
- With Pyrukynd, report signs of sudden red blood cell breakdown or worsening anemia, especially if the medicine was stopped suddenly.
Mitapivat warnings and precautions
▾
- Liver injury (Aqvesme): a boxed warning. It can be serious, usually appears within 6 months, and needs regular liver tests. Avoid in cirrhosis.
- Restricted program: Aqvesme is available only through the Aqvesme REMS, which requires certified prescribers and pharmacies and enrolled patients.
- Acute hemolysis (Pyrukynd): avoid stopping suddenly when possible, because red blood cell breakdown can worsen.
- Early liver injury symptoms can mimic thalassemia symptoms, so report any change.
Mitapivat interactions
▾
Mitapivat is mainly broken down by the CYP3A4 liver enzyme, so drugs affecting that enzyme matter.
- Strong CYP3A inhibitors: raise mitapivat levels and side effect risk. Avoid.
- Moderate CYP3A inhibitors: also raise levels. Avoid with Aqvesme. With Pyrukynd, monitoring and a dose cap apply.
- Strong CYP3A inducers: lower levels and reduce effect. Avoid.
- Moderate CYP3A inducers: lower levels. Alternatives are preferred.
- Hormonal birth control (with Aqvesme): may work less well. Use a non-hormonal backup during use and for 28 days after stopping, or choose another method.
- Other narrow-margin drugs: Aqvesme may change their levels, so monitoring may be needed.
Interactions listed in the FDA-approved label
From the Mitapivat prescribing information (“Drug Interactions”): what the manufacturer studied or reported to the FDA.
- Strong CYP3A inhibitors: Increase mitapivat plasma concentrations, which may increase the risk of adverse reactions. Avoid co-administration.
- Moderate CYP3A inhibitors: Will increase mitapivat plasma concentrations. Avoid co-administration (Aqvesme). For Pyrukynd: monitor Hb and adverse reactions and do not titrate beyond 20 mg twice daily.
- Strong CYP3A inducers: Decrease mitapivat plasma concentrations, which will reduce efficacy. Avoid co-administration.
- Moderate CYP3A inducers: Will decrease mitapivat plasma concentrations. Consider alternative therapies that are not moderate CYP3A inducers. If none, monitor Hb and do not exceed the maximum recommended dose of 100 mg twice daily.
- Sensitive CYP3A substrates including hormonal contraceptives (e.g., ethinyl estradiol): Mitapivat induces CYP3A and will decrease systemic concentrations of these drugs. Avoid with sensitive CYP3A substrates with narrow therapeutic index. Avoid hormonal contraceptives except levonorgestrel intrauterine systems; use an alternative contraceptive not affected by enzyme inducers, or if unavoidable, additional nonhormonal contraception during use and for 28 days after discontinuation.
- CYP2B6 and CYP2C substrates (CYP2C8, CYP2C9, CYP2C19): Mitapivat induces these enzymes in vitro and may decrease systemic concentrations of sensitive substrates. Monitor for loss of therapeutic effect of substrates with narrow therapeutic index.
- UGT1A1 substrates: Mitapivat induces UGT1A1 in vitro and may decrease systemic concentrations of these drugs. Monitor for loss of therapeutic effect of substrates with narrow therapeutic index.
- P-gp substrates: Mitapivat inhibits the P-gp transporter in vitro and may increase systemic concentrations of these drugs. Monitor for adverse reactions of substrates with narrow therapeutic index.
Read the label’s full interactions text
7 DRUG INTERACTIONS Strong CYP3A Inhibitors and Inducers: Avoid concomitant use. ( 7.1 ) Moderate CYP3A Inhibitors: Avoid concomitant use. ( 7.1 ) 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. ( 2.4 , 7.1 ) Sensitive CYP3A substrates including hormonal contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index. ( 7.2 ) CYP2B6, CYP2C and UGT1A1 Substrates: Monitor patients for efficacy of the substrates with narrow therapeutic index. ( 7.2 ) P-gp Substrates: Monitor patients for adverse reactions of the substrates with narrow therapeutic index. ( 7.2 ) 7.1 Effect of Other Drugs on AQVESME Strong CYP3A Inhibitors Clinical Impact Co-administration of AQVESME with strong CYP3A inhibitors increased mitapivat plasma concentrations [see Clinical Pharmacology (12.3) ] . Increased mitapivat plasma concentrations may increase the risks of adverse reactions of AQVESME. Prevention or Management Avoid co-administration of strong CYP3A inhibitors with AQVESME [see Dosage and Administration (2.4) ] . Moderate CYP3A Inhibitors Clinical Impact Co-administration of AQVESME with moderate CYP3A inhibitors will increase mitapivat plasma concentrations [see Clinical Pharmacology (12.3) ] . Prevention or Management Avoid co-administration of moderate CYP3A inhibitors with AQVESME [see Dosage and Administration (2.4) ] . Strong CYP3A Inducers Clinical Impact Co-administration of AQVESME with strong CYP3A inducers decreased mitapivat plasma concentrations [see Clinical Pharmacology (12.3) ] . Decreased mitapivat plasma concentrations will reduce the efficacy of AQVESME. Prevention or Management Avoid co-administration of strong CYP3A inducers with AQVESME [see Dosage and Administration (2.4) ] .
Excerpted — the section continues on the label. Read the full Drug Interactions section on DailyMed →
Not a complete list. The label covers the interactions the manufacturer studied or reported, and our own interaction database does not cover every medicine either. Bring your full medication list, including vitamins and herbal products, to your pharmacist.
Before taking Mitapivat
▾
- Tell your doctor if you have cirrhosis or any liver problems.
- No absolute contraindications are listed.
- Pregnancy: human data are insufficient to assess risk.
- Breastfeeding: no data are available, so discuss benefits and risks.
- Children: safety and effectiveness are not established.
- Adults 65 and older: not enough data to know if response differs.
What Mitapivat does in the body
▾
Mitapivat lowers 2,3-diphosphoglycerate (2,3-DPG) and raises ATP, the cell's energy molecule, in healthy volunteers and in people with thalassemia. At higher-than-recommended doses, it did not meaningfully prolong the QT interval, a measure of heart rhythm timing.
How your body processes Mitapivat
▾
Mitapivat is absorbed quickly, reaching peak levels about 0.5 to 1 hour after a dose, with about 73% absolute bioavailability. A high-fat meal slows absorption but does not change the total amount absorbed. It is highly protein bound and mainly broken down by CYP3A4. Its half-life is about 3 to 5 hours. Most of a dose leaves through urine and feces.
How to store Mitapivat
▾
Store at 20°C to 25°C (68°F to 77°F) with excursions permitted between 15°C and 30°C (59°F and 86°F) . Store the blister wallets in the original carton until use.
Written from the FDA-approved label with AI and reviewed by our pharmacy team. How we use AI →
Supplements & herbs that interact with Mitapivat
154 supplements and herbal products have documented interactions with Mitapivat in the licensed clinical database we use. Most are manageable — but your pharmacist should know about everything you take, including vitamins and herbals.
- Major · 3
- Moderate · 111
- Minor · 40
Educational information from a licensed clinical database (Natural Medicines) — see our data sources & update cadence. Not medical advice: an interaction being documented does not mean it will happen to you; do not start or stop medications or supplements without professional guidance.
Mitapivat: pharmacist answers to common questions
Quick, plain-English answers to the questions patients ask most about Mitapivat — the kind of thing our pharmacy team would talk through with you at the counter.
What is mitapivat for?
▾
How do I take it?
▾
Can I just stop it if I feel fine?
▾
What side effects should I expect?
▾
Why does Aqvesme need so many blood tests?
▾
Does it interact with my other medicines or birth control?
▾
Is Mitapivat available over the counter?
▾
When was Mitapivat first available?
▾
Answers drafted from the FDA-approved label with AI and reviewed by our pharmacy team. How we use AI →
More about Mitapivat on HelloPharmacist
Mitapivat forms and strengths (how it comes)
Mitapivat is available in 1 form. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Tablet
How this form is used
- Pyrukynd treats hemolytic anemia in adults with pyruvate kinase deficiency.
- Aqvesme treats anemia in adults with alpha- or beta-thalassemia.
- Pyrukynd is taken twice daily with or without food, swallowed whole, and tapered rather than stopped suddenly when possible.
- Aqvesme is taken twice daily with or without food, swallowed whole, and needs no taper if stopped.
- Aqvesme needs regular liver tests and is available only through the Aqvesme REMS.
Mitapivat on the U.S. market: products, makers and brands
How Mitapivat appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
1 company lists 7 Mitapivat product records with the FDA, mostly as tablet; the earliest marketing or approval year on record is 2022. Brand names on the directory include Pyrukynd, Aqvesme; the rest are generics.
Also listed with the FDA, not a patient dose form: Kit (8) · Powder (2) — bulk drug substance sold to compounders/manufacturers and multi-item kits. These aren’t counted in the dose-form total above.
How widely Mitapivat is used (Medicaid data)
A general measure of how commonly Mitapivat is prescribed, based on Medicaid — one of the largest public drug programs. The figure below is prescriptions filled in Q1–Q4 2025, summed across every form and brand of the drug.
Think of this as a proxy for how widely Mitapivat is used, drawn from freely available public data. Medicaid is just one program — these numbers don’t include Medicare, other government coverage, or commercial and cash-pay prescriptions, so real-world use is higher than what’s shown here.
How commonly is Mitapivat prescribed? Of the 1,601 medications we measure, Mitapivat ranks #1,484 by Medicaid prescriptions — more than 7% of them.
Utilization by Mitapivat product
Pick a product to see its own Medicaid numbers. Each chip is one clinical product — a specific strength & dosage form as defined by RxNorm — with brand-name and generic versions combined.
Other Mitapivat products — combination packs and listings not yet matched to a specific clinical product (kept visible so every prescription is accounted for)
Summed across the 2 of 8 listed NDC products with Medicaid activity.
Shares are of the Medicaid prescriptions shown here. Product grouping follows RxNorm (the National Library of Medicine’s drug terminology), so “one product” means one strength & dosage form regardless of manufacturer. Dollar figures are gross Medicaid reimbursement before mandatory rebates — rebates (often large, especially for brand-name drugs) are confidential, so actual net cost to Medicaid is lower than shown.
Source: Medicaid State Drug Utilization Data (CMS), aggregated by generic ingredient — summed across every NDC (all brands, strengths, salt forms and dosage forms) of Mitapivat, and across all states and both fee-for-service and managed-care claims. A general popularity signal; it excludes Medicare, commercial insurance and cash prescriptions, so it is not total U.S. use. Based on the 2 of 8 listed Mitapivat NDCs with Medicaid activity.
Compare Mitapivat products
A representative set of FDA-listed product records for Mitapivat — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Tablet | ||||
| 5 mg | Oral | Agios Pharmaceuticals, Inc. × 2 listings | NDA | View NDC → |
| 20 mg | Oral | Agios Pharmaceuticals, Inc. | NDA | View NDC → |
| 50 mg | Oral | Agios Pharmaceuticals, Inc. | NDA | View NDC → |
| 100 mg | Oral | Agios Pharmaceuticals, Inc. | NDA | View NDC → |
| 💊Kits | ||||
| — | — | Agios Pharmaceuticals, Inc. × 2 listings | NDA | View NDC → |
Showing 7 of 7 products — FDA National Drug Code Directory. See every product, package size and price: browse all 7 Mitapivat NDCs →
Mitapivat side effects reported to the FDA (FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Mitapivat — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Mitapivat in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
Mitapivat FDA approval history
How Mitapivat went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
Mitapivat recall history
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
The FDA’s enforcement reports list no Mitapivat recalls since July 2021.
✅No Mitapivat recalls on record since July 2021Source: FDA enforcement (recall) reports, via openFDA; our copy was last updated Sep 24, 2026.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one ingredient concept (IN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). Combination products remain separate concepts with their own pages.
RxCUI 2594468 1 dose form · 4 strengths
-
Dose form (SCDF) Oral Tablet RxCUI 2594472In current FDA listings
Look up RxCUI 2594468 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
Mitapivat supply and shortage status
Whether Mitapivat is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
Mitapivat brand names
Mitapivat is sold under 2 brand names in the FDA directory — Pyrukynd, Aqvesme. A brand and its generic contain the same active ingredient; the brand name belongs to one company's product.
Who makes Mitapivat: manufacturers and labelers
One company lists Mitapivat products with the FDA. By number of product records, the largest are Agios Pharmaceuticals, Inc.. Labelers include manufacturers and repackagers; the product in your bottle depends on which one your pharmacy stocks.
Mitapivat drug class (RxNorm)
Mitapivat belongs to the Pyruvate Kinase Activator class.
Classified by RxNorm RxClass — the U.S. National Library of Medicine's standardized drug-classification service (Established Pharmacologic Class from the FDA, the ATC drug family from the WHO, and mechanism of action). Reference only, not medical advice.
Sources for this Mitapivat page
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How this page is built
HelloPharmacist combines public FDA and NLM records and does not author the underlying clinical facts. The plain-language sections are written with AI from the FDA-approved label text and reviewed by our pharmacy team before publication. Agios Pharmaceuticals, Inc. is the representative DailyMed label linked for verification and dates. This is general education, not medical advice: always talk to your doctor or pharmacist about your own medicines. How we build and review drug pages →