Pyrukynd 20mgx5mg Taper Pack

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 adenosine triphosphate (ATP) production and reduced 2,3-diphosphoglycerate (2,3-DPG) levels, which improves red blood cell survival and reduces hemolysis.

Urgent Side Effects: Seek Medical Help Right Away

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 immediately or seek medical attention:

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 or dizziness
+ Passing out
+ Changes in eyesight
Fast or abnormal heartbeat
Breast pain
Burning, numbness, or tingling sensations that are not normal
Signs of liver problems, such as:
+ Dark urine
+ Tiredness
+ Decreased appetite
+ Upset stomach or stomach pain
+ Light-colored stools
+ Vomiting
+ Yellow skin or eyes (jaundice)

Note: People taking a higher dose of this medication for a different health condition may have a higher risk of liver problems during the first 6 months of treatment.

Other Possible Side Effects

Like all medications, this drug can cause side effects. Many people may not experience any side effects or may only have mild ones. If you notice 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

This is not an exhaustive list of all 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: Important Information to Share with Your Doctor

It is essential to inform your doctor about the following:

Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the symptoms you experienced.
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 be relevant to your treatment with this medication. Therefore, it is vital to discuss all of your medications and health issues with your doctor and pharmacist to ensure safe and effective treatment.

To guarantee your safety, always check with your doctor before starting, stopping, or changing the dose of any medication. This includes prescription and OTC drugs, natural products, and vitamins, as adjusting your treatment regimen without medical supervision can be hazardous.

It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication. Regularly scheduled blood work and laboratory tests, as directed by your doctor, are crucial to monitor your condition.

Do not abruptly discontinue this medication, as this may increase your risk of hemolysis (red blood cell breakdown). If you need to stop taking this drug, your doctor will instruct you on how to gradually taper off the medication. Immediately report any symptoms such as jaundice (yellow skin or eyes), dark urine, dizziness, confusion, fatigue, or shortness of breath to your doctor.

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

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

When taking this medication, birth control pills and other hormone-based contraceptives may be less effective in preventing pregnancy. To minimize the risk of unintended pregnancy, use 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 CYP3A4 inducers (e.g., rifampin, carbamazepine, phenytoin, St. John's wort) - significantly reduce mitapivat exposure.

• Moderate CYP3A4 inducers (e.g., efavirenz, bosentan, etravirine) - may reduce mitapivat exposure, consider dose adjustment or alternative.
• Strong CYP3A4 inhibitors (e.g., ketoconazole, clarithromycin, ritonavir) - significantly increase mitapivat exposure, consider dose reduction of mitapivat.
• P-gp substrates (e.g., digoxin, dabigatran) - mitapivat is a P-gp inducer, may reduce exposure of P-gp substrates.
• CYP3A4 substrates (e.g., midazolam, simvastatin, oral contraceptives) - mitapivat is a moderate CYP3A4 inducer, may reduce exposure of CYP3A4 substrates.

• Moderate CYP3A4 inhibitors (e.g., diltiazem, erythromycin, grapefruit juice) - may increase mitapivat exposure, monitor for adverse effects.
• CYP2B6 substrates (e.g., bupropion) - mitapivat is a weak CYP2B6 inducer, may reduce exposure of CYP2B6 substrates.
• CYP2C8 substrates (e.g., repaglinide) - mitapivat is a weak CYP2C8 inducer, may reduce exposure of CYP2C8 substrates.
• CYP2C9 substrates (e.g., warfarin) - mitapivat is a weak CYP2C9 inducer, may reduce exposure of CYP2C9 substrates, monitor INR.

• Other weak CYP inducers/inhibitors.

• Fatigue
• Pallor
• Jaundice
• Dark urine
• Shortness of breath
• Dizziness
• Headache
• Abdominal pain
• Nausea
• Diarrhea
• Muscle pain
• Back pain
• Rash
• Signs of liver injury (e.g., unexplained nausea, vomiting, abdominal pain, dark urine, yellowing of skin/eyes)

Based on animal studies, mitapivat may cause fetal harm. There are no adequate and well-controlled studies in pregnant women. Use during pregnancy should only be considered if the potential benefit justifies the potential risk to the fetus.

It is not known if mitapivat is excreted in human milk. Mitapivat was detected in the milk of lactating rats. Due to the potential for serious adverse reactions in the breastfed infant, breastfeeding is not recommended during treatment with Pyrukynd and for at least 3 days after the last dose.

The safety and effectiveness of Pyrukynd 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 elderly patients (65 years and older) and younger patients. No specific dose adjustment is required based on age alone.

• Pyrukynd is the first and only FDA-approved oral 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 liver function is crucial, especially during the initial weeks of therapy and after dose adjustments.
• Counsel patients on potential drug interactions, particularly with strong CYP3A4 inducers/inhibitors and P-gp substrates.
• The 'taper pack' containing 20mg and 5mg tablets provides flexibility for precise dose adjustments, which may be useful for initial titration or dose reduction if needed.

• Red blood cell transfusions (supportive care)
• Splenectomy (surgical intervention, often considered for severe cases)
• Folic acid supplementation (to support erythropoiesis)
• Iron chelation therapy (for transfusion-dependent patients to manage iron overload)
• Other investigational therapies for PK deficiency (in clinical trials)

If your symptoms or health issues persist or worsen, it's 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 be sure to check with your pharmacist for more information. If you have any questions or concerns about this 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, including the amount taken and the time it happened, to facilitate prompt and effective treatment.