Risdiplam

What Is Risdiplam?

• Risdiplam (EVRYSDI) is a survival of motor neuron 2 (SMN2) splicing modifier used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.

What are the uses of this medicine?

• This medicine is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.

How does this medicine work?

• Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency.
• Using in vitro assays and studies in transgenic animal models of SMA, risdiplam was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain.

Who Should Not Use this medicine ?

This medicine cannot be used in patients with:

• It is not known if EVRYSDI is safe and effective in children under 2 months of age.

Is this medicine FDA approved?

• It was approved for use in the United States in 2020.

How should this medicine be used?

• EVRYSDI must be constituted by a pharmacist prior to dispensing.

Administration

• EVRYSDI is taken orally once daily after a meal at approximately the same time each day.
• In infants who are breastfed, EVRYSDI should be administered after breastfeeding. EVRYSDI cannot be mixed with formula or milk.
• Instruct patients to drink water after taking EVRYSDI to ensure the drug has been completely swallowed.
• If the patient is unable to swallow and has a nasogastric or gastrostomy tube, EVRYSDI can be administered via the tube. The tube should be flushed with water after delivering EVRYSDI

The recommended dosage is determined by age and body weight as fallows:

• 2 months to less than 2 years of age :0.2 mg/kg
• 2 years of age and older weighing less than 20 kg :0.25 mg/kg
• 2 years of age and older weighing 20 kg or more :5 mg

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Oral Solution: 60 mg of risdiplam as a powder for constitution to provide 0.75 mg/mL solution.

This medicine is available in fallowing brand namesː

• EVRYSDI

What side effects can this medication cause?

Common possible side effects of this medicine include: For later-onset SMA:

• fever
• diarrhea
• rash

For infantile-onset SMA:

• fever
• diarrhea
• rash
• runny nose, sneezing, sore throat, and cough (upper respiratory infection)
• lung infection
• constipation
• vomiting

What special precautions should I follow?

1. Before you start your treatment with EVRYSDI, your healthcare provider may test you for pregnancy.
2. Because EVRYSDI may harm your unborn baby.
3. EVRYSDI may affect a man's ability to have children (fertility).
4. You should receive EVRYSDI from the pharmacy as a liquid that can be given by mouth or through a feeding tube. The liquid solution is prepared by your pharmacist.
5. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement.
6. Avoid getting EVRYSDI on your skin or in your eyes. If EVRYSDI gets on your skin, wash the area with soap and water. If EVRYSDI gets in your eyes, rinse your eyes with water.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are no adequate data on the developmental risk associated with the use of EVRYSDI in pregnant women.

Can this medicine be used in children?

• The safety and effectiveness of EVRYSDI in pediatric patients 2 months of age and older have been established .
• Safety and effectiveness in pediatric patients below the age of 2 months have not been established.

What should I know about storage and disposal of this medication?

• Store the dry powder at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C to 30°C (59°F to 86°F).
• Keep in the original carton.
• Keep the constituted oral solution of EVRYSDI in the original amber bottle to protect from light.
• Store in a refrigerator at 2°C to 8°C (36°F to 46°F)

• Dailymed label info on Risdiplam
• FDA Risdiplam

Risdiplam Resources
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