risdiplam (Rx)

Brand and Other Names:Evrysdi
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Dosing & Uses

AdultPediatric

Dosage Forms & Strengths

powder for oral solution

  • 60mg/bottle

Spinal Muscular Atrophy

Indicated for treatment of spinal muscular atrophy (SMA), including types 1, 2, and 3

5 mg PO qDay

Administer at approximately the same time each day after a meal

Dosage Modifications

Renal impairment

  • Renal impairment is not expected to alter risdiplam exposures

Hepatic impairment

  • Avoid use
  • Safety and efficacy have not been studied in hepatic impairment
  • Hepatic impairment may potentially increase risdiplam exposures

Dosage Forms & Strengths

powder for oral solution

  • 60mg/bottle

Spinal Muscular Atrophy

Indicated for spinal muscular atrophy, including types 1, 2, and 3, in adults and children aged 2 months or older

<2 months: Safety and efficacy not established

≥2 months

  • Administer at approximately the same time each day after a meal or breastfeeding
  • Age ≥2 months to <2 years: 0.2 mg/kg PO qDay
  • Age ≥2 years and weight <20 kg: 0.25 mg/kg PO qDay
  • Age ≥2 years and weight ≥20 kg: 5 mg PO qDay

Dosage Modifications

Renal impairment

  • Renal impairment is not expected to alter risdiplam exposures

Hepatic impairment

  • Avoid use
  • Safety and efficacy have not been studied in hepatic impairment
  • Hepatic impairment may potentially increase risdiplam exposures
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Interactions

Interaction Checker

and risdiplam

No Results

     activity indicator 
    No Interactions Found
    Interactions Found

    Contraindicated

      Serious - Use Alternative

        Significant - Monitor Closely

          Minor

            All Interactions Sort By:
             activity indicator 

            Contraindicated (0)

              Serious - Use Alternative (1)

              • metformin

                risdiplam will increase the level or effect of metformin by decreasing elimination. Avoid or Use Alternate Drug. Risdiplam inhibits MATE1 and MATE2-K. If unable to avoid coadministration with MATE substrates, consider dosage reduction of MATE substrate.

              Monitor Closely (0)

                Minor (0)

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                  Adverse Effects

                  >10%

                  Fever (22%)

                  Diarrhea (17%)

                  Rash (17%)

                  Additional adverse effects in infantile-onset SMA

                  • Upper respiratory tract infection
                  • Pneumonia
                  • Constipation
                  • Vomiting

                  1-10%

                  Mouth and aphthous ulcers (7%)

                  Arthralgia (5%)

                  Urinary tract infection (5%)

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                  Warnings

                  Contraindications

                  None

                  Cautions

                  Drug interaction overview

                  • MATE substrates
                    • Avoid coadministration; if unable to avoid, 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 (eg, metformin)
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                  Pregnancy & Lactation

                  Pregnancy

                  Data are unavailable on developmental risk associated with the use in pregnant females

                  May cause embryofetal harm when administered to pregnant females

                  Pregnancy testing recommended for females of reproductive potential before initiating

                  Animal studies

                  • In animal studies, risdiplam administration 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 exposure

                  Contraception

                  • Females of reproductive potential: Use effective contraception during treatment and for at least 1 month after 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 presence in human milk, effects on breastfed infants, or effects on milk production

                  Orally administered risdiplam was excreted in milk of lactating rats

                  Pregnancy Categories

                  A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.

                  B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk.

                  C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done.

                  D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk.

                  X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist.

                  NA: Information not available.

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                  Pharmacology

                  Mechanism of Action

                  Survival of motor neuron 2 (SMN2) mRNA splicing modifier designed treat mutations in chromosome 5q that lead to SMN protein deficiency

                  Absorption

                  Peak plasma time: 1-4 hr

                  Steady-state reached: 7-14 days

                  Distribution

                  Protein bound: Predominantly bound to serum albumin, without any binding to alpha-1 acid glycoprotein, with a free fraction of 11%

                  Vd: 6.3 L/kg

                  Metabolism

                  Primarily metabolized by flavin monooxygenase 1 and 3 (FMO1 and FMO3) and also by CYPs 1A1, 2J2, 3A4, and 3A7

                  Parent drug was the major component found in plasma, accounting for 83% of drug-related material in circulation

                  Elimination

                  Half-life: ~50 hr

                  Clearance: 2.1 L/h (14.9 kg patient)

                  Excretion: Feces 53% (14% unchanged); urine 28% (8% unchanged)

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                  Administration

                  Oral Preparation

                  Instruct patients or caregivers to prepare the dose using the reusable oral syringe provided

                  Risdiplam must be taken immediately after it is drawn up into the oral syringe; if not taken within 5 minutes, discard the drug from the oral syringe and prepare a new dose

                  Do not mix with formula or milk

                  Preparation of oral solution 0.75 mg/mL by pharmacist

                  • Exercise caution handling the powder for oral solution; avoid inhalation and direct contact with skin or mucous membranes with the dry powder and constituted solution
                  • If such contact occurs, wash thoroughly with soap and water; rinse eyes with water
                  • Wear disposable gloves during preparation and cleanup procedure
                  • Reconstitution
                    1. Gently tap bottom of closed glass bottle to loosen powder
                    2. Remove cap, but do not throw away
                    3. Carefully pour 79 mL of purified water into bottle to yield 0.75 mg/mL oral solution; do not mix with formula or milk
                    4. Insert press-in bottle adapter into bottle opening by pushing it down against bottle lip; ensure it is completely pressed against bottle lip
                    5. Re-cap bottle tightly and shake well for 15 seconds; wait for 10 minutes; solution should appear clear; if not, shake well again for another 15 seconds
                    6. Write expiration date (calculated as 64 days after constitution) and lot number on bottle label; peel off the part of the bottle label listing the powder expiration date
                    7. Put bottle back in its original carton
                    8. Select appropriate oral syringes (6 mL or 12 mL) based on dose and remove other oral syringes from the carton
                    9. 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

                  Oral Administration

                  Take once daily after a meal at approximately the same time each day

                  Drink water after administration to ensure drug is completely swallowed

                  Breastfed infants: Administer after breastfeeding

                  Can be administered via nasogastric or gastrostomy tube; flush tube with water after administering

                  Missed dose

                  • Within 6 hr of missed dose: Take as soon as possible, and then resume usual dosing on following day
                  • >6 hr after missed dose: Skip missed dose and take next dose at regularly scheduled time on the following day
                  • Dose is not fully swallowed or vomited: Do not take another dose to make up for the lost dose; wait until the next day to take at regularly scheduled time

                  Storage

                  Dry powder

                  • Store at 20-25ºC (68-77ºF); excursions permitted to 15-30ºC (59-86ºF)
                  • Keep in the original carton

                  Reconstituted oral solution

                  • Refrigerate at 2-8ºC (36-46ºF)
                  • Do not freeze
                  • Keep oral solution in original amber bottle to protect from light
                  • Keep bottle in an upright position with the cap tightly closed
                  • Discard any unused portion64 days after constitution
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                  Images

                  No images available for this drug.
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                  Patient Handout

                  A Patient Handout is not currently available for this monograph.
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                  Formulary

                  FormularyPatient Discounts

                  Adding plans allows you to compare formulary status to other drugs in the same class.

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                  • View the formulary and any restrictions for each plan.
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                  • Compare formulary status to other drugs in the same class.
                  • Access your plan list on any device – mobile or desktop.

                  The above information is provided for general informational and educational purposes only. Individual plans may vary and formulary information changes. Contact the applicable plan provider for the most current information.

                  Tier Description
                  1 This drug is available at the lowest co-pay. Most commonly, these are generic drugs.
                  2 This drug is available at a middle level co-pay. Most commonly, these are "preferred" (on formulary) brand drugs.
                  3 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs.
                  4 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
                  5 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
                  6 This drug is available at a higher level co-pay. Most commonly, these are "non-preferred" brand drugs or specialty prescription products.
                  NC NOT COVERED – Drugs that are not covered by the plan.
                  Code Definition
                  PA Prior Authorization
                  Drugs that require prior authorization. This restriction requires that specific clinical criteria be met prior to the approval of the prescription.
                  QL Quantity Limits
                  Drugs that have quantity limits associated with each prescription. This restriction typically limits the quantity of the drug that will be covered.
                  ST Step Therapy
                  Drugs that have step therapy associated with each prescription. This restriction typically requires that certain criteria be met prior to approval for the prescription.
                  OR Other Restrictions
                  Drugs that have restrictions other than prior authorization, quantity limits, and step therapy associated with each prescription.
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                  Medscape prescription drug monographs are based on FDA-approved labeling information, unless otherwise noted, combined with additional data derived from primary medical literature.