Pronunciation:

mye-ta-PIV-at

Brand Names:

• Pyrukynd
• Pyrukynd Taper Pack

Dosage Forms:

• Tablet

Uses of This Medicine:

Mitapivat is used to treat hemolytic anemia (low number of red blood cells caused by early breakdown of red blood cells) in patients with pyruvate kinase (PK) deficiency.

This medicine is available only with your doctor's prescription.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of mitapivat in the pediatric population. Safety and efficacy have not been established.

Older adults—

Appropriate studies performed to date have not demonstrated geriatric-specific problems that would limit the usefulness of mitapivat in the elderly.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Alfentanil
• Apalutamide
• Aprepitant
• Atazanavir
• Boceprevir
• Bosentan
• Carbamazepine
• Cenobamate
• Ciprofloxacin
• Clarithromycin
• Cobicistat
• Conivaptan
• Crizotinib
• Cyclosporine
• Dabrafenib
• Desogestrel
• Dienogest
• Digoxin
• Diltiazem
• Dronedarone
• Drospirenone
• Efavirenz
• Enzalutamide
• Erythromycin
• Estetrol
• Estradiol
• Ethinyl Estradiol
• Ethynodiol
• Etonogestrel
• Etravirine
• Fluconazole
• Fluvoxamine
• Fosnetupitant
• Fosphenytoin
• Gestodene
• Idelalisib
• Imatinib
• Indinavir
• Itraconazole
• Ketoconazole
• Letermovir
• Levonorgestrel
• Lopinavir
• Lorlatinib
• Lumacaftor
• Medroxyprogesterone
• Mestranol
• Mitotane
• Modafinil
• Nafcillin
• Nefazodone
• Nelfinavir
• Netupitant
• Nilotinib
• Nomegestrol
• Norelgestromin
• Norethindrone
• Norgestimate
• Norgestrel
• Paclitaxel
• Phenobarbital
• Phenytoin
• Posaconazole
• Primidone
• Rifabutin
• Rifampin
• Ritonavir
• Saquinavir
• Segesterone
• Sirolimus
• St John's Wort
• Telaprevir
• Telithromycin
• Temsirolimus
• Ulipristal
• Verapamil
• Voriconazole
• Warfarin

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following is usually not recommended, but may be unavoidable in some cases. If used together, your doctor may change the dose or how often you use this medicine, or give you special instructions about the use of food, alcohol, or tobacco.

• Grapefruit Juice

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Liver disease, moderate to severe—Use is not recommended in patients with this condition.

Proper Use of This Medicine:

Take this medicine exactly as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered.

This medicine comes with a patient information leaflet. Read and follow the instructions carefully. Ask your doctor or pharmacist if you have any questions.

You may take this medicine with or without food.

Swallow the tablets whole. Do not split, crush, chew, or dissolve it.

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For oral dosage form (tablets):
  • For hemolytic anemia in patients with pyruvate kinase (PK) deficiency:
    • Adults—At first, 5 milligrams (mg) 2 times a day. Your doctor may adjust your dose as needed. However, the dose is usually not more than 50 mg 2 times a day.
    • Children—Use and dose must be determined by your doctor.

Missed dose—

If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

If you miss a dose and it is 4 hours or less, take it as soon as possible, then take your next dose at the normal time. If you miss a dose and it is more than 4 hours, skip the missed dose and take your next dose at the normal time.

Storage—

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Store the tablet in its original container.

Precautions While Using This Medicine:

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 this medicine 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.

Side Effects of This Medicine:

Along with its needed effects, a medicine 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:

More common

Blurred vision

dizziness

headache

nervousness

pounding in the ears

slow or fast heartbeat

Incidence not known

Back pain

chills

clay-colored stools

confusion

dark urine

decreased urination

fever

itching, rash

loss of appetite

nausea

pale skin

stomach pain

trouble breathing

unpleasant breath odor

unusual bleeding or bruising

unusual tiredness or weakness

vomiting of blood

yellow eyes or skin

Some side effects 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:

More common

Breast discomfort

burning, crawling, itching, numbness, prickling, "pins and needles", or tingling feelings

constipation

dry mouth

difficulty in moving

feeling of warmth

muscle pain or stiffness

pain in the joints

pain in the lower back, bottom, hips, or upper legs

redness of the face, neck, arms and occasionally, upper chest

sore throat

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.