Mitapivat Side Effects

Medically reviewed by Drugs.com. Last updated on Mar 15, 2025.

Applies to mitapivat: oral tablet.

Precautions

It is important that your doctor check your progress at regular visits to make sure that this medicine is working properly. Blood tests will be needed to check for unwanted effects.

Do not suddenly Stop taking mitapivat without first checking with your doctor. Your doctor may want you to gradually reduce the amount you are using before stopping it completely. This may help prevent worsening of your condition and reduce the risk of acute hemolysis (rapid breakdown of red blood cells). Call your doctor right away if you have confusion, dark urine, dizziness, trouble breathing, unusual tiredness or weakness, or yellow eyes or skin.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

Serious side effects of mitapivat

Along with its needed effects, mitapivat may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur while taking mitapivat:

Other side effects of mitapivat

Some side effects of mitapivat may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects.

Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

For healthcare professionals

Applies to mitapivat: oral tablet.

Endocrine adverse events

• Very common (10% or more): Estrone decreased in males (56.3%), estradiol decreased in males (12.5%)
• Common (1% to 10%): Blood testosterone increased in males^[Ref]

In 1 study, increases in serum testosterone and decreases in serum estrone and estradiol were observed in men receiving this drug; these hormone changes persisted throughout the study. Reproductive hormone analyses showed decreases in estrone and estradiol to below the lower limit of the reference range and increases in testosterone to above the upper limit of the reference range where baseline was within normal limits. In patients who discontinued this drug and had follow-up hormone measurements, the hormone changes returned close to the baseline levels 28 days after stopping this drug. In female patients, sex hormone analysis was limited due to physiologic variations in hormones during the menstrual cycle and the use of hormonal contraceptives.^[Ref]

Musculoskeletal

• Very common (10% or more): Back pain (included back pain, sciatica, flank pain; 15%), arthralgia (included arthralgia, joint swelling; 10%)
• Frequency not reported: Rib fracture, musculoskeletal pain^[Ref]

Metabolic

• Very common (10% or more): Increased urate (15%)
• Common (1% to 10%): Hypertriglyceridemia (included hypertriglyceridemia, blood triglycerides increased)^[Ref]

Gastrointestinal

• Common (1% to 10%): Gastroenteritis, constipation, dry mouth (included dry mouth, dry lip)^[Ref]

Cardiovascular

• Common (1% to 10%): Hot flush (included hot flush, flushing), hypertension, arrhythmia (included arrhythmia, tachycardia, heart rate increased, atrial fibrillation)
• Frequency not reported: Atrial fibrillation^[Ref]

Respiratory

• Common (1% to 10%): Oropharyngeal pain^[Ref]

Other

• Common (1% to 10%): Breast discomfort^[Ref]

Nervous system

• Common (1% to 10%): Paresthesia^[Ref]

Hematologic

• Frequency not reported: Acute hemolysis, anemia^[Ref]

Acute hemolysis with subsequent anemia was observed after abrupt interruption or discontinuation of this drug in dose-ranging study.^[Ref]

See also:

Further information

Mitapivat side effects can vary depending on the individual. Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Note: Medication side effects may be underreported. If you are experiencing side effects that are not listed, submit a report to the FDA by following this guide.

Medical Disclaimer