Risdiplam
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From MedlinePlus, U.S. National Library of Medicine
Specialty pharmacy: therapy management checklist, storage and cold chain, dosing calendar, Medicare Part D coverage.
Survival of Motor Neuron 2 Splicing Modifier
Dosing in kidney impairment
This label has no separate kidney-impairment dosing section. Check warnings and clinical pharmacology in the full label.
Preparation and administration
Important Administration Instructions It is recommended that a healthcare provider discuss with the patient or caregiver how to prepare the prescribed daily dose prior to administration of the first dose [see Instructions for Use for EVRYSDI for Oral Solution and EVRYSDI Tablets and Patient Information ]. EVRYSDI for Oral Solution In infants who are breastfed, EVRYSDI for oral solution can be administered before or after breastfeeding.
EVRYSDI cannot be mixed with formula or milk. Instruct patients or caregivers to administer the dose using the reusable oral syringe provided. EVRYSDI for oral solution must be taken immediately after it is drawn up into the oral syringe. If EVRYSDI is not taken within 5 minutes, EVRYSDI should be discarded from the oral syringe, and a new dose should be prepared.
Instruct patients to drink water after taking EVRYSDI for oral solution to ensure the drug has been completely swallowed. EVRYSDI for oral solution can be administered via a nasogastric or gastrostomy tube. The tube should be flushed with water after delivering EVRYSDI for oral solution [see Instructions for Use ]. EVRYSDI Tablets Swallow EVRYSDI tablets whole with water.
Do not chew, cut, or crush the tablets. Alternatively, the EVRYSDI tablet can also be dispersed in one teaspoon (5 mL) of room temperature non-chlorinated drinking water (e.g., filtered water). EVRYSDI tablets must not be dispersed in any liquid other than non-chlorinated drinking water. Do not expose the prepared dispersion to sunlight.
Swirl the small cup gently for up to 3 minutes until fully mixed (though some particles will remain). Administer the dispersed tablet immediately. To ensure no particles are left in the small cup, refill it with at least one tablespoon (15 mL) of non-chlorinated drinking water, swirl, and administer immediately again. EVRYSDI must be taken immediately after it is dispersed in non-chlorinated drinking water.
Discard the prepared dispersion if it is not used within 10 minutes of adding non-chlorinated drinking water. The dispersed EVRYSDI tablet can be administered via a nasogastric or gastrostomy tube that is 8 French or higher. Flush the tube with the non-chlorinated drinking water [at least one tablespoon (15 mL)] used to rinse the dispersion cup [see Instructions for Use ].
Preparation of Powder for Oral Solution by Healthcare Provider EVRYSDI powder must be constituted to the oral solution by a pharmacist or other healthcare provider prior to dispensing to the patient. Preparation of the EVRYSDI Oral Solution 0.75 mg/mL The EVRYSDI " Instructions for Constitution " booklet contains more detailed instructions on the preparation of the oral solution [see Instructions for Constitution ] .
Caution should be exercised when handling EVRYSDI powder for oral solution . Avoid inhalation and direct contact with skin or mucous membranes with the dry powder and the constituted solution. If such contact occurs, wash thoroughly with soap and water; rinse eyes with water. Wear disposable gloves during the preparation and cleanup procedure.
Gently tap the bottom of the closed glass bottle to loosen the powder. Remove the cap. Do not throw away the cap. Carefully pour 79 mL of purified water into the EVRYSDI bottle to yield the 0.75 mg/mL oral solution. Do not mix EVRYSDI with formula or milk. Insert the press-in bottle adapter into the bottle opening by pushing it down against the bottle lip.
Ensure it is completely pressed against the bottle lip. Re-cap the bottle tightly and shake well for 15 seconds. Wait for 10 minutes. You should have obtained a clear solution. If not, shake well again for another 15 seconds or until you have obtained a clear solution. Write the date of expiration of the constituted oral solution (calculated as 64 days after constitution) and the lot number on the bottle label.
Peel off the part of the bottle label that has the expiration date of the powder. Put the bottle back in its original carton. Select the appropriate oral syringes (1 mL, 6 mL, or 12 mL) based on the patient's dosage and remove the other oral syringes from the carton. Dispense with the " Instructions for Use " and FDA-approved patient labeling.
Alert patients to read the important handling information described in the Instructions for Use . Storage 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). Do not freeze. Discard any unused portion 64 days after constitution.
Keep the bottle in an upright position with the cap tightly closed. If refrigeration is not available, EVRYSDI can be kept at room temperature up to 40°C (up to 104°F) for a combined total of 5 days. EVRYSDI can be removed from, and returned to, a refrigerator. The total combined time out of refrigeration should not exceed 5 days.
Drug interactions
Check interactions
Add the patient's other drugs. Matches come from both FDA labels: by name, drug class, and enzyme or transporter pathway.
- Risdiplam
Label text (2 paragraphs)
Avoid coadministration with drugs that are substrates of multidrug and toxin extrusion (MATE) transporters. ( 7.1 )
7.1 Effect of EVRYSDI on Substrates of Multidrug and Toxin Extrusion (MATE) Protein Transporters Based on in vitro data, EVRYSDI may increase plasma concentrations of drugs eliminated via MATE1 or MATE2-K [see Clinical Pharmacology (12.3) ] , such as metformin. Avoid coadministration of EVRYSDI with MATE substrates. If coadministration cannot be avoided, monitor for drug-related toxicities and consider dosage reduction of the coadministered drug (based on the labeling of that drug) if needed.
Contraindications
None. None. ( 4 )
Safety data
Reported adverse events (FAERS)
Liver injury (LiverTox)
ELikelihood score E: Unlikely cause of clinically apparent liver injury.
Risdiplam is a small molecular modifier of RNA splicing of survival motor neuron 2 protein used in the therapy of spinal muscular atrophy, a rare progressive neuromuscular disorder that typically leads to disability and death within the first two years of life. Risdiplam is given orally once daily and has not been linked to serum aminotransferase or bilirubin elevations during therapy nor to instances of clinically apparent liver injury.
ABCDEA: well known cause; E: unlikely cause. [HD]: high doses only.
Pregnancy and lactation
Pregnancy
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.
Data Animal Data Oral administration of risdiplam (0, 1, 3, or 7.5 mg/kg/day) to pregnant rats throughout organogenesis resulted in decreased fetal body weights and increased incidences of fetal structural variations at the highest dose tested, which was not associated with maternal toxicity. The no-effect level for adverse effects on embryofetal development (3 mg/kg/day) was associated with maternal plasma exposure (AUC) approximately 2 times that in humans at the maximum recommended human dose (MRHD) of 5 mg.
Oral administration of risdiplam (0, 1, 4, or 12 mg/kg/day) to pregnant rabbits throughout organogenesis resulted in embryofetal mortality, fetal malformations (hydrocephaly), and structural variations at the highest dose tested, which was associated with maternal toxicity. The no-effect dose for adverse effects on embryofetal development (4 mg/kg/day) was associated with maternal plasma exposure (AUC) approximately 4 times that in humans at the MRHD.
When risdiplam (0, 0.75, 1.5, or 3 mg/kg/day) was orally administered to rats throughout pregnancy and lactation, gestation was prolonged in the dams, and delayed sexual maturation (vaginal opening) and impaired reproductive function (decreased numbers of corpora lutea, implantation sites, and live embryos) were observed in female offspring at the highest dose. The no-effect dose for adverse effects on pre- and postnatal development in rats (1.5…
For breastfeeding, also see LactMed (National Library of Medicine), which summarizes milk levels and infant effects.
Supply and cost
Drug shortage status
Recalls (last 24 months)
Therapeutic equivalence (Orange Book)
Price ladder
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