pharmacopeia
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/api/v1/drug/risdiplam

Mechanism of action

Sourced from openFDA

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.

Multidrug and Toxin Extrusion Transporter 1Multidrug and Toxin Extrusion Transporter 2 K

Indications

Sourced from openFDA
  • EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. EVRYSDI is a survival of motor neuron 2 (SMN2) splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Contraindications

Sourced from openFDA
  • None. None.contraindicated

Dosage & administration

Sourced from openFDA

Administer once daily with or without food per the table below ( 2.1 ): Age and Body Weight Recommended Daily Dosage Dosage Form Less than 2 months of age 0.15 mg/kg EVRYSDI for Oral Solution 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 EVRYSDI for Oral Solution or EVRYSDI Tablet Swallow EVRYSDI tablet whole with water or dispersed in non-chlorinated drinking water (e.g., filtered water). ( 2.2 ) Administer EVRYSDI for oral solution with the provided oral syringe. ( 2.2 ) EVRYSDI for oral solution must be constituted by a healthcare provider prior to dispensing. ( 2.4 ) See Full Prescribing Information for important preparation and administration instructions. ( 2.2 , 2.4 ) 2.1 Dosing Information EVRYSDI is administered orally once daily with or without food at approximately the same time each day. The recommended dosage is determined by age and body weight (see Table 1 ). EVRYSDI tablets are available for patients prescribed the 5 mg dose.

Adverse reactions

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The most common adverse reactions in later-onset SMA (incidence at least 10% of patients treated with EVRYSDI and more frequent than control) were fever, diarrhea, and rash. ( 6.1 ) The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions with an incidence of at least 10% were upper respiratory tract infection, lower respiratory tract infection, constipation, vomiting, and cough. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genentech at 1-888-835-2555 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in clinical trials of another drug and may not reflect the rates observed in practice. In clinical trials including patients with infantile-onset SMA, later-onset SMA, and pre-symptomatic SMA, a total of 491 patients (51% female, 74% Caucasian) were exposed to EVRYSDI for up to a median duration of 48.1 months (range: 0.6 to 63.4 months), with 231 patients receiving treatment for more than 24 months. At the time of first EVRYSDI dose, 90 (18%) patients were 18 years and older, 119 (24%) were 12 years to less than 18 years, 189 (39%) were 2 years to less than 12 years, 67 (14%) 2 months to less than 2 years, and 26 (5%) were less than 2 months.

Use in specific populations

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Pregnancy: Based on animal data, may cause fetal harm. ( 8.1 ) 8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy and fetal/neonatal/infant outcomes in women exposed to EVRYSDI during pregnancy. Physicians are encouraged to register patients and pregnant women are encouraged to register themselves by calling 1-833-760-1098 or visiting https://www.evrysdipregnancyregistry.com. Risk Summary There are no adequate data on the developmental risk associated with the use of EVRYSDI in pregnant women. In animal studies, administration of risdiplam during pregnancy or throughout pregnancy and lactation resulted in adverse effects on development (embryofetal mortality, malformations, decreased fetal body weights, and reproductive impairment in offspring) at or above clinically relevant drug exposures [see Data ]. The estimated background risk of major birth defects and miscarriage for the indicated populations is unknown. In the U.S. general population, the estimated background risk of major birth defect and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Based on animal data, advise pregnant women of the potential risk to the fetus.

Pharmacokinetics

Sourced from openFDA
Metabolism
Pharmacokinetics of EVRYSDI have been characterized in healthy adult subjects and in patients with SMA. After administration of EVRYSDI as an oral solution, pharmacokinetics of risdiplam were approximately linear between 0.6 and 18 mg in a single-ascending-dose study in healthy adult subjects, and between 0.02 and 0.25 mg/kg once daily in a multiple-ascending-dose study in patients with SMA.

Approval history

Sourced from openFDA
  • Aug 7, 2020NDANDA213535Genentech Inc
  • Feb 11, 2025NDANDA219285Genentech Inc

FAERS reports

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Reference statistics only. FAERS reports are voluntarily submitted and are not incidence rates, safety signals, or causal evidence. Counts reflect reporting volume — how often a reaction was reported, not how often it occurs. For decision-grade use, consult openFDA and the FAERS Public Dashboard directly.
3,960 total reports matchedLatest report Share = reports listing the reaction ÷ total matched reports. Rows can sum to >100% because a single report often lists multiple reactions.
  1. 1No Adverse Event3388.5%
  2. 2Diarrhoea2917.3%
  3. 3Asthenia2195.5%
  4. 4Pneumonia2105.3%
  5. 5Death1944.9%
  6. 6Pyrexia1754.4%
  7. 7Fatigue1553.9%
  8. 8Muscular Weakness1423.6%
  9. 9Vomiting1363.4%
  10. 10Drug Ineffective1303.3%
  11. 11Headache1092.8%
  12. 12Rash1092.8%
  13. 13Covid-191072.7%
  14. 14Nausea1002.5%
  15. 15Product Dose Omission Issue932.3%

Clinical trials

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The 10 most recently updated of 32 ClinicalTrials.gov registrations naming Risdiplam as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

Frequently asked questions

How does Risdiplam 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.
What is Risdiplam used for?
According to FDA labeling, Risdiplam carries indications including: EVRYSDI is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. EVRYSDI is a survival of motor neuron 2 (SMN2) splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.
What class of drug is Risdiplam?
Risdiplam is classified as Other drugs for disorders of the musculo-skeletal system, Survival of Motor Neuron 2 Splicing Modifier, Gene Editing or Modification, Multidrug and Toxin Extrusion Transporter 1 Inhibitors, Multidrug and Toxin Extrusion Transporter 2 K Inhibitors, Survival of Motor Neuron 2 Splicing Modifiers, Central Nervous System Stimulation, Increased Protein Synthesis.
What are the brand names for Risdiplam?
Risdiplam is marketed under brand names including Evrysdi.
What are the contraindications for Risdiplam?
Risdiplam labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.
Note. Data for risdiplam is illustrative MVP content compiled from public sources. pharmacopeia is for educational and informational use only and is not a substitute for professional medical advice.

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