Pyrukynd is a brand-name oral tablet prescribed to treat low red blood cell counts caused by pyruvate kinase deficiency (PKD). Pyrukynd contains the active ingredient mitapivat and belongs to the PK activator drug class.

Specifically, the Food and Drug Administration (FDA) has approved Pyrukynd to treat hemolytic anemia in adults with PKD. This is a genetic condition that causes premature hemolysis (early breakdown) of red blood cells. For details about PKD and hemolytic anemia, see the “Pyrukynd uses” section.

Drug details

You’ll find key information about Pyrukynd below.

  • Drug form: oral tablet
  • Generic available? no
  • Prescription required? yes
  • Controlled substance? no
  • Year of FDA approval: 2022
  • Accelerated approval? no

Pyrukynd is available only as a brand-name medication. It’s not currently available in generic form.

A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics usually cost less than brand-name drugs.

The Pyrukynd dosage your doctor prescribes will depend on several factors. These include:

  • the severity of the condition you’re taking Pyrukynd to treat
  • other medical conditions you may have
  • other medications you may take

The following information describes the typical dosage that’s commonly prescribed or recommended. However, be sure to take the dosage your doctor prescribes for you. Your doctor will determine the best dosage to fit your needs.

Drug form and strengths

Pyrukynd comes as an oral tablet in three strengths:

  • 5 milligrams (mg)
  • 20 mg
  • 50 mg

Dosage for hemolytic anemia

Pyrukynd is prescribed to treat hemolytic anemia in adults with pyruvate kinase deficiency. (See “Pyrukynd uses” for details about this condition.)

The typical starting dosage is 5 mg twice per day. After you’ve taken this dosage for 4 weeks, your doctor will check your hemoglobin (Hb) level.

If your Hb level is within the normal range and you have not needed a blood transfusion in the past 8 weeks, your doctor will keep your dosage at 5 mg twice per day. If your Hb level is below the normal range and you have needed a transfusion, your doctor will increase your dosage to 20 mg twice per day.

After another 4 weeks, your doctor will check your Hb level again.

If your Hb level is within the normal range and you have not needed a transfusion, your doctor will have you continue with your current maintenance dosage (5 mg or 20 mg twice per day, according to your doctor’s instructions).

If your Hb level is below the normal range or you have needed a transfusion, your doctor will increase your dosage to 50 mg twice per day. This is the maximum recommended dosage of Pyrukynd.

About taking Pyrukynd

Below you’ll find information about key dosage issues.

  • When to take: You’ll take Pyrukynd twice per day, such as in the morning and in the evening. Taking the medication around the same times every day helps keep a steady level of the drug in your body. This helps Pyrukynd work effectively.
  • If you miss a dose: If you miss a dose by 4 hours or less, take the dose as soon as possible. If it’s been more than 4 hours, skip the missed dose. Then take your next scheduled dose and continue your usual dosing schedule.
  • Taking Pyrukynd with food: You can take Pyrukynd with or without food.
  • Crushing, splitting, or chewing Pyrukynd: You should swallow Pyrukynd tablets whole. Do not split, cut, crush, chew, or dissolve the tablets.
  • Length of use: Pyrukynd is meant to be a long-term treatment. If you and your doctor determine that Pyrukynd is safe and effective for you, you’ll likely take it long term. It’s important that you do not suddenly stop taking or pause Pyrukynd, as this can lead to harmful effects. For more information, see the “Pyrukynd precautions” section.

Pyrukynd can cause mild or serious side effects. The following lists contain some of the key side effects that may occur while taking Pyrukynd. These lists do not include all possible side effects.

For more information about the possible side effects of Pyrukynd, talk with your doctor or pharmacist. They can give you tips on how to manage any side effects that may be concerning or bothersome.

Note: The Food and Drug Administration (FDA) tracks side effects of drugs it has approved. Sharing your experience helps make medications safer for everyone by giving doctors and researchers more information about how the drug works in real life. If you develop a side effect while taking Pyrukynd and want to tell the FDA about it, visit MedWatch or call 800-FDA-1088.

Mild side effects

Below is a partial list of mild side effects of Pyrukynd. To learn about other mild side effects, talk with your doctor or pharmacist or view the drug’s prescribing information.

Mild side effects of Pyrukynd can include:

Most of these side effects may go away within a few days to a couple of weeks. However, if they become more severe or don’t go away, talk with your doctor or pharmacist.

* Sex and gender exist on spectrums. Use of the term “male” in this article refers to sex assigned at birth.

Serious side effects

Serious side effects from Pyrukynd aren’t common, but they can occur. Call your doctor right away if you have serious side effects. Call 911 or your local emergency number if your symptoms feel life threatening or you think you’re having a medical emergency.

Serious side effects and their symptoms can include:

ALLERGIC REACTION

For some people, Pyrukynd can cause an allergic reaction, but this side effect wasn’t reported in the drug’s studies.

In general, symptoms of allergic reaction can be mild or serious.

Ways to manage

For mild allergic reaction symptoms, such as a mild rash, call your doctor right away. They may recommend treatments to help manage your symptoms. They’ll also let you know whether you should keep taking the medication.

For severe allergic reaction symptoms, such as swelling or trouble breathing, call 911 or your local emergency number right away. These symptoms require immediate medical care because they can become life threatening. If you’ve had a serious allergic reaction to Pyrukynd, your doctor may recommend taking a different medication instead.

The FDA approves prescription drugs such as Pyrukynd to treat certain conditions. Pyrukynd may also be used off-label for other conditions. Off-label drug use is when an FDA-approved drug is prescribed for a purpose other than what it’s approved for.

Pyrukynd for hemolytic anemia caused by pyruvate kinase deficiency

Pyrukynd is FDA approved to treat hemolytic anemia caused by pyruvate kinase deficiency (PKD). This is a rare, inherited condition that causes premature hemolysis (early breakdown) of red blood cells (RBCs).

Healthy RBCs circulate through your bloodstream for about 120 days before hemolysis. Your body makes new RBCs continuously to maintain your levels.

Pyruvate kinase (PK) is an enzyme that converts glucose (sugar) into energy. When you have PKD, the RBC form of PK is mutated. As a result, RBCs don’t have enough energy, and this causes an abnormal cell shape. Your body destroys these abnormal RBCs earlier and more quickly than your body can replenish them, leading to anemia (a low RBC count).

PKD can range from mild to severe. For instance, the condition may be serious from birth or may not cause problems until adulthood.

Signs and symptoms of PKD and hemolytic anemia can include:

Pyrukynd increases PK activity, which helps prevent premature RBC hemolysis. This helps raise RBC levels and reduces symptoms. To learn more about how Pyrukynd works, see the section “Common questions about Pyrukynd.”

Pyrukynd and children

Pyrukynd is not approved for any use in children. It’s not known whether the drug is safe or effective in children.

Pyrukynd can interact with several other medications and possibly certain supplements.

Before taking Pyrukynd, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, and supplements you take. Sharing this information can help you avoid potential interactions.

Interactions with medications and supplements

Below is a list of medications and supplements that can interact with Pyrukynd. This list does not contain all drugs that may interact with Pyrukynd. If you have questions about drug interactions that may affect you, ask your doctor or pharmacist.

Medications that can interact with Pyrukynd• strong CYP3A inhibitors, such as erythromycin (E.E.S., EryTab) and itraconazole (Sporanox, Tolsura)
• moderate CYP3A inhibitors, such as fluconazole (Diflucan)
• strong CYP3A inducers, such as rifampin (Rifadin)
• moderate CYP3A inducers, such as efavirenz (Sustiva)
• CYP3A substrates, such as midazolam (Nayzilam)
• CYP2B6 and CYP2C substrates, such as warfarin (Jantoven), omeprazole, and hormonal birth control
• UGT1A1 substrates, such as etoposide and other anticancer drugs
• P-gp substrates, such as digoxin and loperamide (Imodium)
Vitamins or supplements that can interact with Pyrukyndpossibly the herb St. John’s wort, which is a strong CYP3A inducer

CYP3, CYP2, UGT1A1, and P-gp are proteins in your body that are involved in metabolizing (breaking down) natural substances and drugs, including Pyrukynd. Taking Pyrukynd with another drug that interacts with this process may cause atypical levels of Pyrukynd or the other drug. Depending on the specific drug, this may increase your risk of side effects or decrease the effectiveness of Pyrukynd or the other drug.

Your doctor or pharmacist can tell you whether other medications or supplements you take may interact with Pyrukynd. They’ll also recommend what to do about potential interactions, such as switching the other medication for a different drug or monitoring you more closely for side effects.

Alcohol interaction

Alcohol is not known to interact with Pyrukynd. However, Pyrukynd can cause liver damage as a side effect. Heavy alcohol consumption may also damage your liver, so drinking alcohol during Pyrukynd treatment may increase this risk.

If you have questions about how much alcohol is safe to drink while taking Pyrukynd, talk with your doctor or pharmacist.

As with all medications, the cost of Pyrukynd can vary. The actual price you’ll pay depends on your insurance plan, your location, and the pharmacy you use.

Financial and insurance assistance: If you need financial support to pay for Pyrukynd or need help understanding your insurance coverage, help is available.

A program called myAgios is available for Pyrukynd. The drug manufacturer (Agios) also offers a Pyrukynd Copay Program for people who have commercial insurance. For more information and to find out whether you’re eligible for support, call 800-951-3889 or visit the program website.

Generic version: Pyrukynd is not available in a generic form. A generic drug is an exact copy of the active ingredient in a brand-name medication. Generics tend to cost less than brand-name drugs.

Learn more about saving money on prescriptions.

A few other drugs are available that can treat your condition, but none of them are similar to Pyrukynd. If you’re interested in finding an alternative to Pyrukynd, talk with your doctor. They can tell you about other medications that may work well for you.

Other drugs that may be prescribed for hemolytic anemia include:

If you can become pregnant, consider the following information about pregnancy, birth control, and breastfeeding.

Pyrukynd and pregnancy

It’s not known whether Pyrukynd should be taken during pregnancy. But there are risks involved in having untreated pyruvate kinase deficiency (PKD) while pregnant. (Pyrukynd is used to treat hemolytic anemia caused by PKD.)

If you’re planning a pregnancy or can become pregnant, talk with your doctor about potential treatments for PKD during pregnancy. Together, you can consider the risks and benefits of your treatment options and decide whether you should take Pyrukynd during pregnancy.

Pyrukynd and birth control

It’s not known whether Pyrukynd is safe to take during pregnancy. If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control needs during Pyrukynd treatment.

Pyrukynd and breastfeeding

It’s not known whether Pyrukynd should be taken while breastfeeding. If you’re currently breastfeeding or planning to do so, talk with your doctor before taking this medication.

Here are answers to some frequently asked questions about Pyrukynd.

How does Pyrukynd work?

Pyrukynd works by increasing the activityTrusted Source of an enzyme called pyruvate kinase (PK). An inherited deficiency in the red blood cell (RBC) form of PK can cause hemolytic anemia, a condition in which RBCs break down more quickly than your body replenishes them, leading to anemia (a low RBC count).

PK helps produce energy inside RBCs. When you have PK deficiency (PKD), your RBCs don’t have enough energy. Your body removes these abnormal RBCs from your bloodstream in a process called hemolysis. By increasing PK activity, Pyrukynd increases energy production. This increases the level of normal RBCs and reduces symptoms of PKD.

For more information about your condition, see the “Pyrukynd uses” section. For more information about how Pyrukynd works, talk with your doctor.

Will I still need blood transfusions with Pyrukynd treatment?

Possibly. Blood transfusions are a common treatment for hemolytic anemia. In clinical trials, some people who took Pyrukynd needed fewer transfusions than people who took a placebo (no active ingredient). This effect may be long lasting, as some people remained transfusion-free for up to 21 months in a long-term study.

The severity of PKD and hemolytic anemia varies from person to person. Depending on the severity of your anemia, you may still need blood transfusions to manage your RBC levels and symptoms.

Before taking Pyrukynd, discuss your health history with your doctor. Pyrukynd may not be right for you if you have certain medical conditions or other factors affecting your health. Be sure to talk with your doctor if any of the following apply to you:

  • liver problems
  • previous allergic reaction to this or a similar drug
  • pregnancy
  • breastfeeding

Stopping Pyrukynd

You should take Pyrukynd exactly as your doctor prescribes. Do not suddenly stop taking Pyrukynd or pause treatment. Suddenly stopping or pausing Pyrukynd can lead to rapid destruction of red blood cells (RBCs) and acute hemolytic anemia. A sudden breakdown of RBCs can overwhelm your body.

Symptoms of acute hemolytic anemia can include:

Tell your doctor right away if you develop any of these symptoms.

If you’re interested in stopping Pyrukynd treatment, talk with your doctor first. They’ll recommend how to do so safely. For instance, they’ll taper (slowly reduce) your Pyrukynd dose over time. For more information about this, talk with your doctor or pharmacist.

Note: For more information about the potential negative effects of Pyrukynd, see the “Pyrukynd side effects” section.

Disclaimer: Medical News Today has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.