Generic name: risdiplam [ ris-DIP-lam ]
Drug class: Miscellaneous uncategorized agents
Medically reviewed by Drugs.com. Last updated on Mar 27, 2023.
Uses for risdiplam
Risdiplam is used to treat spinal muscular atrophy, a rare nerve and muscle disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to early death.
This medicine is available only with your doctor's prescription.
Before using risdiplam
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:
Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of risdiplam in children (newborn infants and older). Safety and efficacy have been established.
Appropriate studies on the relationship of age to the effects of risdiplam have not been performed in the geriatric population.
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Interactions with Medicines
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
Interactions with Food/Tobacco/Alcohol
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.
Other Medical Problems
The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:
- Liver disease—Use with caution. The effects may be increased because of slower removal of the medicine from the body.
Proper use of risdiplam
Take this medicine only as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered.
This medicine should come with a patient information leaflet. Read and follow these instructions carefully. Ask your doctor if you have any questions.
Measure the liquid medicine with the oral syringe and take it within 5 minutes. Throw away any unused medicine after 5 minutes. Do not save leftover medicine.
Take the medicine after a meal at about the same time each day. Drink a glass of water after taking the liquid to make sure it has been swallowed completely.
If you are giving this medicine to an infant, give it after breastfeeding. Do not mix it with milk or formula.
If you or your child cannot swallow the medicine, you may take it using a nasogastric or gastrotomy tube. Flush the tube with water after to make sure you get all of the medicine.
The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.
The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.
- For oral dosage form (solution):
- For spinal muscular atrophy:
- Adults and children 2 years of age and older weighing 20 kilograms (kg) or more—5 milligrams (mg) once a day.
- Children 2 years of age and older weighing less than 20 kg—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.25 milligram per kilogram (mg/kg) of body weight once a day.
- Infants 2 months to less than 2 years of age—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.2 mg/kg of body weight once a day.
- Infants younger than 2 months of age—Dose is based on body weight and must be determined by your doctor. The dose is usually 0.15 mg/kg of body weight once a day.
- For spinal muscular atrophy:
If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.
If you miss a dose and it is within 6 hours of your usual dose schedule, take it as soon as possible, and then go back to your regular schedule. If you miss a dose and it is more than 6 hours of your usual dose schedule, skip the missed dose and take your next dose at the regular time.
Store in the refrigerator. Do not freeze.
Keep out of the reach of children.
Do not keep outdated medicine or medicine no longer needed.
Ask your healthcare professional how you should dispose of any medicine you do not use.
Throw away any unused medicine 64 days after it was mixed.
Precautions while using risdiplam
It is very important that your doctor check your child's progress at regular visits to make sure this medicine is working properly. Blood tests may be needed to check for unwanted effects.
Using this medicine while you are pregnant can harm your unborn baby. If you are a woman who can bear children, your doctor may give you a pregnancy test before you start using this medicine to make sure you are not pregnant. Use an effective form of birth control during treatment and for at least 1 month after the last dose.
If you plan to have children, talk with your doctor before using this medicine. Some men using this medicine have become infertile (unable to have children).
Do not take other medicines unless they have been discussed with your doctor. This includes prescription or non-prescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.
Side Effects of risdiplam
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.
Check with your doctor immediately if any of the following side effects occur:
- Body aches or pain
- chest pain or tightness
- ear congestion
- loss of voice
- runny or stuffy nose
- sore throat
- trouble breathing
- unusual tiredness or weakness
- Bladder pain
- bloody or cloudy urine
- difficult, burning, or painful urination
- frequent urge to urinate
- lower back or side pain
Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:
- Canker sores
- skin rash
- sores, ulcers, or white spots on the lips or tongue or inside the mouth
- Difficulty in moving
- joint pain
- muscle pain or stiffness
Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
More about risdiplam
- Check interactions
- Compare alternatives
- Side effects
- Dosage information
- During pregnancy
- Drug class: miscellaneous uncategorized agents
- En español
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Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.