Risdiplam

Risdiplam is a prescription medication used for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older.

• Risdiplamis are available under the following different brand names: Evrysdi

Adult and pediatric dosage

Powder for oral solution

• 60mg/bottle

Spinal Muscular Atrophy

Adult dosage

• 5 mg orally once a day
• Administer at approximately the same time each day after a meal

Pediatric dosage

• Children below 2 months: Safety and efficacy not established
• Children above 2 months
• Administer at approximately the same time each day after a meal or breastfeeding
• Age above 2 months to children below 2 years: 0.2 mg/kg orally once a day
• Age above 2 years and weight below 20 kg: 0.25 mg/kg orally once a day
• Age above 2 years and weight above 20 kg: 5 mg orally once a day

Dosage Considerations – Should be Given as Follows: 

See “Dosages”

Common side effects of Risdiplam include:

• fever,
• diarrhea,
• rash,
• upper respiratory tract infection,
• pneumonia,
• constipation, and
• vomiting

Serious side effects of Risdiplam include:

• hives,
• difficult breathing,
• swelling of the face, lips, tongue, or throat,
• pain or burning while urinating, and
• signs of a lung infection--fever, cough with mucus, chest pain, feeling short of breath

Rare side effects of Risdiplam include:

• none 

This is not a complete list of side effects and other serious side effects or health problems that may occur as a result of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Risdiplam has severe interactions with no other drugs.
• Risdiplam has serious interactions with no other drugs.
• Risdiplam has moderate interactions with the following drugs:
  • metformin
• Risdiplam has minor interactions with no other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all your products. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your health care professional or doctor for additional medical advice, or if you have health questions or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Risdiplam?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Risdiplam?”

Cautions

• Drug interaction overview
• MATE substrates
• Avoid coadministration; if unable to avoid it, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug (based on the labeling of that drug) if needed
• Based on in vitro data, risdiplam may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K (e.g., metformin)

Pregnancy & Lactation

• Data are unavailable on the developmental risk associated with the use in pregnant females
• It May causes embryofetal harm when administered to pregnant females
• Pregnancy testing is recommended for females of reproductive potential before initiating
• Contraception
  • Females of reproductive potential: Use effective contraception during treatment and for at least 1 month after the last dose
• Infertility
  • Male fertility may be compromised by treatment
  • Counsel male patients of reproductive potential about potential reproductive effects
  • Male patients may consider sperm preservation before treatment
• Lactation
  • Data are not available on the presence of human milk, effects on breastfed infants, or effects on milk production
  • Orally administered risdiplam was excreted in the milk of lactating rats