Risdiplam Tablets (Evrysdi) is an SMN2 splicing modifier used for the treatment of adult and pediatric patients with spinal muscular atrophy (SMA). As a breakthrough drug in the field of gene therapy, its use must strictly follow the age- and weight-based graded dosing regimen.
Indications for Risdiplam Tablets (Evrysdi)
Core Treatment Scope
Risdiplam is indicated for the treatment of genetically confirmed spinal muscular atrophy.
Pre-symptomatic SMA: Infants who have not yet developed clinical symptoms (first dose administered at ≤ 6 weeks of age).
Infantile SMA (Type 1): Symptoms appear between 28 days and 3 months of age.
Late-onset SMA (Type 2 or 3): Symptoms appear after 18 months of age.
Usage Restrictions
Dosing regimen: Must be administered strictly according to age and weight stratification.
Contraindications: No absolute contraindications are listed in the current package insert.
Special monitoring: Regular assessment of motor function milestone progression is required.
Specifications and Properties of Risdiplam Tablets (Evrysdi)
Basic Parameters
Dosage form: Oral solution (requires reconstitution) and film-coated tablets.
Tablet strength: 5mg per tablet (pale yellow, round, biconvex tablets, engraved with "EVR" on one side).
Oral solution strength: 60mg per vial (after reconstitution, it becomes a yellow-green solution with a concentration of 0.75mg/mL).
Formulation Components
Active ingredient: Risdiplam.
Tablet excipients: Colloidal silicon dioxide, crospovidone, mannitol, etc.
Solution excipients: Ascorbic acid, disodium edetate, isomalt, etc.
Dosage Form Characteristics
Tablets: Can be swallowed whole or dissolved in non-chlorinated drinking water (e.g., filtered water).
Oral solution: Must be administered using a dedicated oral syringe and must not be mixed with formula milk/milk.
Storage Methods for Risdiplam Tablets (Evrysdi)
Unreconstituted Powder
Temperature: Store at room temperature (15-30℃) in accordance with USP room temperature control regulations.
Light protection requirement: Keep in the original packaging.
Expiration date: Refer to the label on the vial.
Reconstituted Solution
Refrigerated storage: Store upright at 2-8℃ (must be used within 64 days).
Room temperature stability: Can be stored at a temperature not exceeding 40℃ for a total of 5 days.
Light protection requirement: Must be kept in the original amber vial.
Tablet Storage
Routine conditions: Store at 20-25℃ (short-term fluctuations between 15-30℃ are allowed).
Moisture-proof measure: Keep the bottle cap tightly closed.
Dispersion: Prepare and use immediately (administer within 10 minutes).
