Pyrukynd 50mgx20mg Taper Pack

Mitapivat is an allosteric activator of pyruvate kinase (PK). It binds to and activates wild-type and mutated PK enzymes, increasing PK activity in red blood cells. This leads to increased ATP production and reduced levels of 2,3-bisphosphoglycerate (2,3-BPG), which improves red blood cell survival and reduces hemolysis in patients with pyruvate kinase deficiency.

Urgent Side Effects: Seek Medical Attention Immediately

Although rare, some people may experience severe and potentially life-threatening side effects while taking this medication. If you notice any of the following symptoms, contact your doctor or seek medical help right away:

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
Fast or abnormal heartbeat
Breast pain
Burning, numbness, or tingling sensations that are not normal

Additionally, people taking a higher dose of this medication for a different health condition may have an increased risk of liver problems during the first 6 months of treatment. If you experience any of the following symptoms, contact your doctor immediately:

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. While 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 or seek medical help:

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

This is not an exhaustive list of possible side effects. If you have questions or concerns about side effects, 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 any 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 (OTC) 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.
Please note that this is not an exhaustive list of all potential drug interactions or health problems that may affect the use of this medication.

To ensure your safety, it is vital to inform your doctor and pharmacist about all your medications, health problems, and any potential interactions. Before starting, stopping, or changing the dose of any medication, including this one, consult with your doctor to confirm that it is safe to do so.

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 symptoms such as jaundice (yellowing of the skin or eyes), dark urine, dizziness, confusion, fatigue, or shortness of breath to your doctor.

Grapefruit and grapefruit juice should be avoided while taking this medication, as they 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. It is also important to note that birth control pills and other hormone-based contraceptives may be less effective while taking this medication. To prevent pregnancy, consider using an additional form of birth control, such as a condom.

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

• Strong CYP3A inducers (e.g., rifampin, carbamazepine, enzalutamide, mitotane, phenytoin, St. John's Wort)

• Moderate CYP3A inducers (e.g., efavirenz, bosentan, etravirine, modafinil, nafcillin): Avoid concomitant use.
• Strong CYP3A inhibitors (e.g., ketoconazole, clarithromycin, ritonavir, itraconazole, indinavir, nelfinavir, saquinavir, telithromycin): Reduce mitapivat dose.

• Sensitive CYP3A substrates (e.g., midazolam, oral contraceptives, simvastatin): Mitapivat is a moderate CYP3A inducer; may decrease exposure of co-administered CYP3A substrates. Consider alternative or increased dose of CYP3A substrate.
• P-glycoprotein (P-gp) substrates (e.g., digoxin): Monitor for increased exposure of P-gp substrates.

• Signs and symptoms of hemolytic anemia (e.g., fatigue, pallor, jaundice, dark urine, shortness of breath)
• Signs and symptoms of adverse reactions (e.g., headache, nausea, insomnia, arthralgia, back pain, rash)
• Signs of potential drug interactions

There are no adequate and well-controlled studies of mitapivat in pregnant women. Animal studies showed adverse developmental effects (e.g., reduced fetal body weight, skeletal variations) at exposures higher than clinical exposure. Pyrukynd should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. A pregnancy exposure registry is available.

There are no data on the presence of mitapivat in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Pyrukynd and any potential adverse effects on the breastfed infant from Pyrukynd or from the underlying maternal condition.

Safety and effectiveness in pediatric patients have not been established. Pyrukynd is not indicated for use in pediatric patients.

No overall differences in safety or effectiveness were observed between patients 65 years of age and older and younger patients, but greater sensitivity of some older individuals cannot be ruled out. No specific 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.
• The 'Taper Pack' is designed to facilitate the initial dose titration, starting at 5 mg BID, then 20 mg BID, before reaching the maintenance dose of 50 mg BID.
• Patients should be educated on the importance of adherence to the twice-daily dosing schedule.
• Close monitoring for drug interactions, especially with CYP3A inducers/inhibitors, is crucial.
• Patients should be advised to report any new or worsening symptoms of hemolysis or adverse effects.

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

If your symptoms or health issues persist or worsen, it is essential to contact your doctor for further guidance. To ensure your safety, 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. If you have any questions or concerns about this medication, don't hesitate to discuss them with 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 detailed information about the overdose, including the substance taken, the amount, and the time it occurred.