Mitapivat

Mitapivat is used to treat hemolytic anemia (a blood disorder that occurs when red blood cells are destroyed faster than they can be made in the body) in people with pyruvate kinase deficiency. Mitapivat is in a class of medications called pyruvate kinase activators. It works by increasing pyruvate kinase available to increase red blood cell (RBC) lifespan.

Mitapivat comes as a tablet to take by mouth. It is usually taken with or without food 2 times a day. Take mitapivat at around the same times every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take mitapivat exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the tablets/capsules whole; do not split, chew, or crush them.

Your doctor will probably start you on a low dose of mitapivat and gradually increase your dose, typically every 4 weeks.

Mitapivat controls hemolytic anemia from pyruvate kinase deficiency but does not cure it. Continue to take mitapivat even if you feel well. Do not stop taking mitapivat without talking to your doctor. If you suddenly stop taking mitapivat, you may experience symptoms of acute hemolysis and anemia (yellowing of the skin or whites of the eyes, dark urine, dizziness, confusion, tiredness, difficulty breathing). Your doctor will probably decrease your dose gradually.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

Before taking mitapivat,

• tell your doctor and pharmacist if you are allergic to mitapivat, any other medications, or any of the ingredients in mitapivat tablet. Ask your pharmacist for a list of the ingredients.
• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take. Be sure to mention any of the following: carbamazepine (Carbatrol, Epitol, Equetro, Tegretol, Teril); digoxin (Lanoxin); diltiazem (Cardizem, Cartia XT, Taztia XT, Tiazac); efavirenz (Sustiva; in Atripla, in Symfi); erythromycin (E.E.S., ERY-C, Ery-Tab, Erythrocin); fluconazole (Diflucan); hormonal contraceptives containing ethinyl estradiol, itraconazole (Sporanox), ketoconazole (Nizoral), nevirapine (Viramune); oxcarbazepine (Oxtellar XR, Trileptal); phenobarbital; phenytoin (Dilantin, Phenytek); rifabutin (Mycobutin, in Talicia); rifampin (Rifadin, Rimactane); and verapamil (Calan, Verelan).Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while taking mitapivat, call your doctor.

Unless your doctor tells you otherwise, continue your normal diet.

Take the missed dose as soon as you remember it if it is within 4 hours of the usual dosing time. If more than 4 hours have passed, skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one.

Mitapivat may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• back pain
• joint pain
• nausea, vomiting, diarrhea or stomach pain
• hot flushes (warmth and redness of face, neck, chest or back)
• sore throat
• breast pain or swelling
• constipation
• dry mouth
• rapid or fast heart beat
• tingling or prickly feeling in hands, arms, feet or legs

Mitapivat may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom). Mitapivat tablets should stay in their original blister packaging until ready to use.

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. https://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (https://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to mitapivat.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.

• Pyrukynd^®