Risdiplam
Spinal Muscular Atrophy
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Drug Status
Government Approvals
US(FDA), UK(BNF)
WHO Essential Medicine
NO
Known Teratogen
No
Pharmaceutical Class
Survival of Motor Neuron 2 Splicing Modifier
Controlled Drug Substance
No
Risdiplam
Summary
Risdiplam is used to treat a genetic disorder called Spinal Muscular Atrophy (SMA) in both children and adults. SMA leads to muscle weakness and atrophy due to the loss of motor neurons.
Risdiplam works by modifying the splicing of the SMN2 gene. This increases the production of a functional protein called SMN which is essential for motor neuron survival. By increasing SMN protein levels, Risdiplam helps improve motor function and slows the progression of SMA.
Risdiplam is taken orally once daily after a meal. The dosage varies based on age and body weight. For adults and children aged 2 years and older weighing 20 kg or more, the recommended dose is 5 mg once daily. For children aged 2 months to less than 2 years, the dose is 0.2 mg/kg. For those aged 2 years and older weighing less than 20 kg, the dose is 0.25 mg/kg.
Common side effects of Risdiplam include fever, diarrhea, and rash. In infantile-onset SMA, common side effects include upper and lower respiratory tract infections, constipation, vomiting, and cough. Serious adverse effects are rare but can include cutaneous vasculitis.
Risdiplam may cause harm to the fetus, so women of childbearing potential should use effective contraception during treatment and for at least one month after the last dose. It should not be used in patients with hypersensitivity to any of its components. Patients should be monitored for drug interactions, especially with MATE substrates.
Indications and Purpose
How does Risdiplam work?
Risdiplam works by increasing the production of a protein called SMN, which is essential for muscle health. This helps improve motor function in people with spinal muscular atrophy, which is a genetic disorder affecting muscle strength. Think of it like boosting the body's ability to support muscle function, leading to better movement and strength over time.
Is Risdiplam effective?
Risdiplam is effective for treating spinal muscular atrophy, which is a genetic disorder affecting muscle strength. Clinical studies show that Risdiplam improves motor function in patients with this condition. It works by increasing the production of a protein essential for muscle health. Regular monitoring by your doctor is important to assess the effectiveness of Risdiplam for your condition.
What is Risdiplam?
Risdiplam is a medication used to treat spinal muscular atrophy, which is a genetic disorder affecting muscle strength. It works by increasing the production of a protein essential for muscle health. Risdiplam is taken orally and is used as a long-term treatment to improve motor function in patients with this condition. Always follow your doctor's instructions when taking Risdiplam.
Directions for Use
For how long do I take Risdiplam?
Risdiplam is usually a long-term medication for managing spinal muscular atrophy, which is a chronic condition. You'll typically take Risdiplam every day as a lifelong treatment unless your doctor suggests otherwise. How long you'll need this medication depends on your body's response and any side effects you experience. Always talk with your doctor before changing or stopping your Risdiplam treatment.
How do I dispose of Risdiplam?
Dispose of unused Risdiplam by taking it to a drug take-back program or collection site at a pharmacy or hospital. If you can't find a take-back program, you can throw it in the trash at home. First, take it out of its original container, mix it with something undesirable like used coffee grounds, seal the mixture in a plastic bag, and throw it away.
How do I take Risdiplam?
Risdiplam is usually taken once daily. Follow your doctor's instructions on the exact timing and whether to take it with food. Do not crush or mix it with food or liquids unless advised by your doctor. If you miss a dose, take it as soon as you remember unless it's almost time for your next dose. In that case, skip the missed dose. Never take two doses at once. Always follow your doctor's specific advice about diet and fluid intake while taking this medication.
How long does it take for Risdiplam to start working?
Risdiplam begins working in your body shortly after you take it, but noticeable improvements in muscle function may take several weeks to months. The time it takes to see full benefits can vary based on individual factors like age and overall health. Regular check-ups with your doctor are important to monitor your progress and adjust your treatment as needed.
How should I store Risdiplam?
Store Risdiplam at room temperature, away from moisture and light. Keep it in its original container, tightly closed, and out of reach of children. Don't store it in humid places like bathrooms. Check the expiration date regularly and properly dispose of any unused or expired medication. Follow any specific storage instructions provided by your pharmacist or doctor.
What is the usual dose of Risdiplam?
The usual starting dose of Risdiplam for adults is determined by your doctor based on your specific condition. It's typically taken once daily. Your doctor may adjust the dose based on your response and any side effects. Always follow your doctor's specific dosing instructions. For children or the elderly, dose adjustments may be necessary, and careful monitoring is required.
Warnings and Precautions
Can I take Risdiplam with other prescription drugs?
Risdiplam may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K transporters, such as metformin. It is advised to avoid coadministration with MATE substrates. If unavoidable, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug. Risdiplam is a weak inhibitor of CYP3A, but no significant interactions with CYP3A substrates are expected.
Can Risdiplam be taken safely while breastfeeding?
Risdiplam is not recommended while breastfeeding. Limited information is available on whether it passes into human breast milk. If you're taking Risdiplam and want to breastfeed, talk with your doctor about safer medication options. Your doctor can help determine the best treatment plan for you and your baby.
Can Risdiplam be taken safely while pregnant?
Risdiplam is not recommended during pregnancy due to potential risks to the unborn baby. Limited information is available on its use in pregnant women. If you're pregnant or planning to become pregnant, talk with your doctor about the safest way to manage your condition. Your doctor can help create a treatment plan that protects both you and your baby.
Does Risdiplam have adverse effects?
Adverse effects are unwanted reactions to a medication. Risdiplam can cause side effects, but most people tolerate it well. Common adverse effects include nausea and diarrhea. Serious side effects are rare but require immediate medical attention. Always inform your doctor about any new or worsening symptoms while taking Risdiplam. Your doctor can help determine if these symptoms are related to the medication.
Does Risdiplam have any safety warnings?
Risdiplam has important safety warnings. It can cause serious side effects, so it's crucial to follow your doctor's instructions. Not adhering to safety warnings can lead to severe health issues. If you experience any unusual symptoms or side effects, contact your healthcare provider immediately. Regular monitoring by your doctor is important to ensure safe use of Risdiplam.
Is it safe to drink alcohol while taking Risdiplam?
It's best to avoid alcohol while taking Risdiplam. Alcohol can interfere with the medication's effectiveness and may increase the risk of side effects. If you choose to drink occasionally, limit your alcohol intake and watch for any unusual symptoms. Talk with your doctor about alcohol use while taking Risdiplam to get personalized advice based on your health situation.
Is it safe to exercise while taking Risdiplam?
You can exercise while taking Risdiplam, but be mindful of your body's response. Risdiplam can cause fatigue, which might affect your exercise capacity. To exercise safely, listen to your body and rest if you feel tired. Most people can maintain their regular exercise routine while taking Risdiplam, but check with your doctor if you have concerns.
Is it safe to stop Risdiplam?
Stopping Risdiplam suddenly can cause problems for your health condition. Always talk with your doctor before stopping Risdiplam. They might suggest gradually reducing your dose or switching to a different medication to keep your condition under control. Your doctor will help you make any medication changes safely to protect your health.
Is Risdiplam addictive?
Risdiplam is not addictive or habit-forming. It doesn't cause dependency or withdrawal symptoms when you stop taking it. Risdiplam works by affecting specific proteins in the body, not brain chemistry, so it doesn't lead to addiction. If you have concerns about medication dependence, you can feel confident that Risdiplam doesn't carry this risk.
Is Risdiplam safe for the elderly?
Clinical studies of Risdiplam did not include patients aged 65 years and older to determine whether they respond differently from younger adult patients. Therefore, no specific dosage adjustments are recommended for elderly patients. However, as with any medication, elderly patients should use Risdiplam under the guidance of a healthcare provider, who will consider their overall health and any other medications they may be taking.
What are the most common side effects of Risdiplam?
Side effects are unwanted reactions to a medication. Common side effects of Risdiplam include nausea and diarrhea. These effects vary from person to person. If you notice new symptoms after starting Risdiplam, they might be temporary or unrelated to the medication. Talk with your doctor before stopping any medication. Your doctor can help determine if the side effects are related to Risdiplam.
Who should avoid taking Risdiplam?
Don't take Risdiplam if you're allergic to it or its ingredients. Serious allergic reactions require immediate medical help. Risdiplam is not recommended during pregnancy or breastfeeding due to potential risks to the baby. Always consult your doctor about any concerns or conditions that might affect the use of Risdiplam. Your doctor will help determine if Risdiplam is safe for you.
