Pyrukynd 5mg Tablets

Mitapivat is an oral, small molecule allosteric activator of pyruvate kinase (PK). It works by binding to and activating the mutated pyruvate kinase enzyme in patients with pyruvate kinase deficiency, leading to increased PK activity, increased ATP levels, and reduced 2,3-diphosphoglycerate (2,3-DPG) levels, which improves red blood cell metabolism and reduces hemolysis.

Urgent Side Effects: Seek Medical Help Right Away

While rare, some people may experience severe and potentially life-threatening side effects when taking this medication. If you notice any of the following symptoms, contact your doctor or seek medical attention immediately:

Signs of an allergic reaction, such as:
+ Rash
+ Hives
+ Itching
+ Red, swollen, blistered, or peeling skin with or without fever
+ Wheezing
+ Tightness in the chest or throat
+ Trouble breathing, swallowing, or talking
+ Unusual hoarseness
+ Swelling of the mouth, face, lips, tongue, or throat
Signs of high blood pressure, including:
+ Severe headache
+ Dizziness
+ Fainting
+ Changes in eyesight
Abnormal or rapid heartbeat
Breast pain
Unusual burning, numbness, or tingling sensations
Signs of liver problems (more common in people taking higher doses for a different health condition), such as:
+ Dark urine
+ Fatigue
+ Decreased appetite
+ Upset stomach or stomach pain
+ Light-colored stools
+ Vomiting
+ Yellow skin or eyes

Other Possible Side Effects

Like all medications, this drug can cause side effects. Although many people may not experience any side effects or only minor ones, it's essential to discuss any concerns with your doctor. If you experience any of the following side effects or any other symptoms that bother you or do not go away, contact your doctor:

Back pain
Joint pain
Stomach pain or diarrhea
Flushing
Mouth or throat pain or irritation
Constipation
Dry mouth
Dry lips

Reporting Side Effects

This list is not exhaustive, and you may experience other side effects. If you have questions or concerns, contact your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.

Before taking this medication, it is essential to inform your doctor about the following:

Any allergies you have, including allergies to this drug, its components, or other substances. Be sure to describe the allergic reaction you experienced, including the symptoms that occurred.
If you have liver disease, as this may affect the safety and efficacy of the medication.
Any medications you are currently taking, including prescription and over-the-counter drugs, natural products, and vitamins. This is crucial because certain medications, such as those used to treat HIV, infections, seizures, and other conditions, may interact with this drug and should not be taken concurrently.
It is important to note that this is not an exhaustive list of all potential drug interactions or health problems that may be relevant to your treatment with this medication.

To ensure your safety, it is vital to provide your doctor and pharmacist with a comprehensive list of all your medications, including prescription and over-the-counter drugs, natural products, and vitamins, as well as any health problems you are experiencing. This will enable them to assess potential interactions and determine whether it is safe for you to take this medication. Never start, stop, or modify the dose of any medication without first consulting your doctor.

It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. Regular blood tests and other laboratory assessments should be conducted as directed by your doctor to monitor your condition.

To minimize the risk of hemolysis (red blood cell breakdown), do not abruptly discontinue this medication. If you need to stop taking this drug, your doctor will instruct you on how to gradually taper off the dosage. Immediately report any signs of hemolysis to your doctor, including jaundice (yellowing of the skin or eyes), dark urine, dizziness, confusion, fatigue, or shortness of breath.

Avoid consuming grapefruit and grapefruit juice while taking this medication, as it may interact with the drug. Additionally, be aware that this medication can cause elevated triglyceride levels. If you have a history of high triglyceride levels, inform your doctor.

In men, this medication may decrease levels of the reproductive hormones estrone and estradiol. If you have concerns or questions, discuss them with your doctor.

Women taking this medication should be aware that birth control pills and other hormone-based contraceptives may be less effective. To prevent pregnancy, use an additional form of birth control, such as a condom, while taking this medication.

If you are pregnant, plan to become pregnant, or are breastfeeding, consult your doctor to discuss the potential benefits and risks of this medication to you and your baby.

• Strong CYP3A inducers (e.g., rifampin, carbamazepine, phenytoin, St. John's Wort): Concomitant use is not recommended as it can significantly decrease mitapivat exposure and efficacy.
• Strong CYP3A inhibitors (e.g., ketoconazole, itraconazole, clarithromycin, ritonavir): Concomitant use is not recommended as it can significantly increase mitapivat exposure and adverse reactions.

• Moderate CYP3A inducers (e.g., bosentan, efavirenz, etravirine, modafinil): May decrease mitapivat exposure. Consider dose adjustment or alternative agents.
• Moderate CYP3A inhibitors (e.g., erythromycin, diltiazem, verapamil, fluconazole): May increase mitapivat exposure. Monitor for adverse reactions.
• CYP3A substrates (e.g., midazolam, simvastatin, oral contraceptives): Mitapivat is a moderate CYP3A inducer. May decrease exposure of co-administered CYP3A substrates, reducing their efficacy. Consider dose increase for the substrate or alternative agents.
• P-gp substrates (e.g., digoxin, dabigatran): Mitapivat is a P-gp inducer. May decrease exposure of co-administered P-gp substrates. Monitor or adjust dose of P-gp substrate.

• Signs and symptoms of hemolysis (e.g., fatigue, pallor, jaundice, dark urine)
• Signs and symptoms of adverse reactions (e.g., nausea, headache, diarrhea, arthralgia, rash)
• Signs and symptoms of liver dysfunction (e.g., unexplained nausea, vomiting, abdominal pain, fatigue, anorexia, dark urine, jaundice)

There are no adequate and well-controlled studies of Pyrukynd in pregnant women. Animal studies have shown adverse developmental effects (embryo-fetal mortality, reduced fetal weight, skeletal variations) at exposures higher than clinical exposure. Use during pregnancy only if the potential benefit justifies the potential risk to the fetus.

It is not known whether mitapivat is excreted in human milk. Due to the potential for serious adverse reactions in breastfed infants, breastfeeding is not recommended during treatment with Pyrukynd and for at least 3 days after the last dose.

Safety and effectiveness in pediatric patients have not been established.

No overall differences in safety or effectiveness were observed between elderly patients (65 years and older) and younger patients, but greater sensitivity of some older individuals cannot be ruled out. No dose adjustment is required based on age.

• Pyrukynd is the first and only FDA-approved therapy for hemolytic anemia in adults with pyruvate kinase deficiency.
• Patients should be educated on the importance of adherence to the twice-daily dosing regimen.
• Close monitoring of hemoglobin levels and transfusion requirements is crucial to assess treatment response.
• Counsel patients on potential drug interactions, especially with strong CYP3A inducers/inhibitors and P-gp substrates.
• Patients should be advised to report any signs of liver dysfunction immediately.

• Supportive care (e.g., red blood cell transfusions)
• Splenectomy (surgical removal of the spleen, which can reduce hemolysis but carries risks)
• Iron chelation therapy (for iron overload due to chronic transfusions)
• Folic acid supplementation (to support erythropoiesis)

If your symptoms or health issues persist or worsen, it is essential to contact your doctor promptly. To ensure safe and effective treatment, never share your medication with others or take someone else's medication. Some medications may come with an additional patient information leaflet, so it's a good idea to check with your pharmacist for more information. If you have any questions or concerns about your medication, don't hesitate to reach out to your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately call your local poison control center or seek emergency medical attention. When seeking help, be prepared to provide details about the medication taken, the amount, and the time it happened.