Pyrukynd (mitapivat)
An approved treatment for Pyruvate Kinase Deficiency.
The same compound appears under different names depending on the context. Here is how to identify Mitapivat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Mitapivat
- Brand name
- Pyrukynd
- Development code
- AG-348
- Drug class
- Pyruvate kinase activator
- Manufacturer
- Agios Pharmaceuticals
- How it's taken
- A tablet taken twice a day with or without food, swallowed whole.
A twice-daily pill, approved February 17, 2022, that treats hemolytic anemia in adults with pyruvate kinase (PK) deficiency. It was the first drug approved for PK deficiency. The same drug is sold under a different brand, Aqvesme, for thalassemia.
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Where Mitapivat fits
The only drug approved for PK deficiency, used to raise hemoglobin and cut transfusions in adults, on top of supportive care.
How Mitapivat works
In PK deficiency a weak pyruvate kinase enzyme leaves red blood cells short of energy, so they are destroyed early. Mitapivat binds the enzyme and switches it on harder, raising the cells' energy supply and slowing their breakdown. It works only when some enzyme is made: people with 2 variants that make no enzyme at all were left out of its main trial because they did not respond in earlier testing.
Mechanism: Oral activator of the red blood cell enzyme pyruvate kinase that raises the cells' energy (ATP) supply so fewer break down early
Side effects and safety
- Red cell breakdown if stopped suddenly. Acute hemolysis followed abrupt stops in a dose-ranging study. Taper the dose rather than stopping, and watch for yellow eyes, dark urine, dizziness, tiredness or shortness of breath.
- Liver injury seen in another condition. Liver tests before starting and monthly for the first 6 months. The drug is paused if ALT rises above 5 times normal and stopped if liver injury from it is suspected.
- Hemoglobin and transfusion needs before each dose increase
- Liver tests before starting, then monthly for 6 months
- Signs of red cell breakdown when doses are lowered or stopped
Pyrukynd has no boxed warning. Stopping it suddenly can cause rapid red cell breakdown, so it is tapered off. Because liver injury was seen with a higher dose in another condition, liver tests are checked before starting and monthly for the first 6 months. In the ACTIVATE trial, back pain (15% against 8% on placebo) and joint pain (10% against 5%) were the most common side effects, and uric acid rose in 15%. In men, testosterone rose and estrone and estradiol fell, returning close to baseline about 28 days after stopping.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Mitapivat
A tablet taken twice a day with or without food, swallowed whole. It starts at 5 mg twice a day and can rise every 4 weeks to 20 mg and then 50 mg twice a day, checking hemoglobin and transfusion needs first, because some people do well at a lower dose. It is stopped if there is no benefit by 24 weeks, and always tapered rather than stopped suddenly.
Availability and cost
Only available as the brand-name product.
Help paying for Pyrukynd
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Adults with PK deficiency who have commercial or private insurance can pay $0 per prescription when their copay or coinsurance is higher than that. People on Medicare Part D, Medicaid, Medigap, TRICARE, VA, DoD or state-funded programs cannot use it. People with only Medicare Part A and B and no Part D drug coverage can. You have to qualify again each calendar year.
For: private insurance · source - Free medicine program
Uninsured and underinsured adults with PK deficiency who meet financial and other rules get Pyrukynd at no cost for up to a full calendar year. You must reapply each year and have a home address in the US or Puerto Rico. The program ends once you have insurance coverage and the out-of-pocket cost is affordable.
For: no insurance, underinsured · source - Bridge or quick-start supply
The Coverage Interruption Program is for adults with commercial insurance who are already on Pyrukynd, have had at least one fill and lose or change coverage for a while. They get a free 28-day prescription with up to 2 refills. People in government or state health programs cannot use it.
For: private insurance · source - Insurance and case manager help
The program is free. A Patient Support Manager checks your insurance coverage and out-of-pocket costs, works with your doctor on insurance requirements and goes over which financial help programs you may qualify for. Support is also available in Spanish.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: No dollar cap is stated for the copay program. Hours are Monday to Friday, 8 AM to 8 PM ET.
Access and eligibility
Approved for adults with PK deficiency; safety and effectiveness in children have not been established. People with 2 PKLR variants that make no working enzyme, or who are homozygous for the R479H variant, were excluded from ACTIVATE because they did not respond in earlier testing.
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
ACTIVATE (NCT03548220) enrolled 80 adults not on regular transfusions: 40% on Pyrukynd had a hemoglobin response, a sustained rise of at least 1.5 g/dL, against 0% on placebo, with an average change of 1.7 g/dL against a fall of 0.1. ACTIVATE-T (NCT03559699), a single-group trial in 27 regularly transfused adults, found 33% cut transfusions by at least a third and 22% became transfusion-free; all 6 stayed transfusion-free in a long-term extension.
Development history
Developed by Agios Pharmaceuticals as AG-348. Approved February 17, 2022 as Pyrukynd for PK deficiency in adults, the first drug for the condition. The FDA approved the same drug as Aqvesme for thalassemia on December 23, 2025, at a higher dose and with a REMS program for liver monitoring, and is due to decide on sickle cell disease by November 1, 2026. Pediatric trials in PK deficiency (ACTIVATE-Kids and ACTIVATE-KidsT) are active but no longer recruiting.
Explore Pyruvate Kinase Deficiency trials
Common questions about Mitapivat
▸What is Mitapivat (Pyrukynd)?
A twice-daily pill, approved February 17, 2022, that treats hemolytic anemia in adults with pyruvate kinase (PK) deficiency. It was the first drug approved for PK deficiency. The same drug is sold under a different brand, Aqvesme, for thalassemia.
▸How does Mitapivat work?
In PK deficiency a weak pyruvate kinase enzyme leaves red blood cells short of energy, so they are destroyed early. Mitapivat binds the enzyme and switches it on harder, raising the cells' energy supply and slowing their breakdown. It works only when some enzyme is made: people with 2 variants that make no enzyme at all were left out of its main trial because they did not respond in earlier testing.
▸What are the side effects of Mitapivat?
Pyrukynd has no boxed warning. Stopping it suddenly can cause rapid red cell breakdown, so it is tapered off. Because liver injury was seen with a higher dose in another condition, liver tests are checked before starting and monthly for the first 6 months. In the ACTIVATE trial, back pain (15% against 8% on placebo) and joint pain (10% against 5%) were the most common side effects, and uric acid rose in 15%. In men, testosterone rose and estrone and estradiol fell, returning close to baseline about 28 days after stopping.
▸How is Mitapivat taken?
A tablet taken twice a day with or without food, swallowed whole. It starts at 5 mg twice a day and can rise every 4 weeks to 20 mg and then 50 mg twice a day, checking hemoglobin and transfusion needs first, because some people do well at a lower dose. It is stopped if there is no benefit by 24 weeks, and always tapered rather than stopped suddenly.
▸Is Mitapivat FDA approved?
Yes, Mitapivat (Pyrukynd) is FDA approved (2022) for the treatment of Pyruvate Kinase Deficiency.
▸Is Pyrukynd the same drug as Aqvesme?
Yes, both are mitapivat from Agios. Pyrukynd is the brand for PK deficiency, started at 5 mg twice a day and raised up to 50 mg twice a day. Aqvesme is the brand for thalassemia at 100 mg twice a day, with a boxed liver warning and a REMS program. They are not interchangeable without a new prescription.
More: Aqvesme for thalassemia
▸Can children take Pyrukynd?
Not yet. Pyrukynd is approved for adults; the label says safety and effectiveness in children have not been established. Agios's pediatric trials, ACTIVATE-Kids (NCT05175105) and ACTIVATE-KidsT (NCT05144256), are active but no longer recruiting.
▸Why can't Pyrukynd be stopped suddenly?
Stopping it abruptly caused rapid red blood cell breakdown and anemia in a dose-ranging study. The label calls for tapering the dose down when stopping, and watching for yellow eyes, dark urine, dizziness or shortness of breath.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2026-06. PYRUKYND (mitapivat) tablets: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=4ccee896-e313-4c6c-9674-b4746ada0a90
- ClinicalTrials.gov. ACTIVATE: mitapivat in adults with PK deficiency not regularly transfused. https://clinicaltrials.gov/study/NCT03548220
- ClinicalTrials.gov. ACTIVATE-T: mitapivat in regularly transfused adults with PK deficiency. https://clinicaltrials.gov/study/NCT03559699