Risdiplam

What it is and how it works

Risdiplam modifies splicing of SMN2 pre-mRNA to promote inclusion of exon 7, increasing production of functional full-length SMN protein. This compensates for the deficient SMN protein caused by SMN1 mutations in spinal muscular atrophy.

Indications

5q spinal muscular atrophy in patients with a clinical diagnosis of types 1, 2 or 3 or with one to four SMN2 copies, according to the product licence.

Dosage and administration (summary)

Taken orally once daily after a meal at about the same time each day, using the oral syringe provided; dose depends on age and body weight (for patients aged 2 years and over weighing 20 kg or more, 5 mg once daily).

Contraindications

Hypersensitivity to risdiplam.

Warnings and precautions

Embryofetal toxicity has been shown in animals, so effective contraception is needed during treatment and for at least one month after in women of childbearing potential; male fertility may be impaired. Avoid contact of the solution with skin and eyes.

Pregnancy

Not recommended in pregnancy because of embryofetal toxicity in animal studies.

Storage and supply

Store the powder below 25 °C; after reconstitution, store the oral solution at 2–8 °C, protected from light, and use within the period stated by the manufacturer.

Source: reference summary prepared from the EU SmPC/EPAR, BNF and WHO Model Formulary. The approved product information in Iran governs.