Formulary
Risdiplam: Indications, Dosing, Side Effects and Interactions
Risdiplam is a survival motor neurone 2 (SMN2) pre-mRNA splicing modifier that increases the production of SMN protein, thereby helping to compensate for the...
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Risdiplam: Indications, Dosing, Side Effects and Interactions
Risdiplam is a survival motor neurone 2 (SMN2) pre-mRNA splicing modifier that increases the production of SMN protein, thereby helping to compensate for the...
Drug action
Risdiplam is a survival motor neurone 2 (SMN2) pre-mRNA splicing modifier that increases the production of SMN protein, thereby helping to compensate for the defect in the SMN1 gene found in 5q spinal muscular atrophy.
Indications and dose
**5q spinal muscular atrophy (initiated by a specialist)**
- **Adult** (By Mouth): 5 mg once daily, to be taken after a meal at the same time each day.
**5q spinal muscular atrophy (initiated by a specialist) for risdiplam**
- **Neonate** (By mouth): 150 micrograms/kg once daily, to be taken after a feed at the same time each day, dose based on corrected age for preterm infants.
- **Child 1 month** (By mouth): 150 micrograms/kg once daily, to be taken after a feed at the same time each day, dose based on corrected age for preterm infants.
- **Child 2-23 months** (By mouth): 200 micrograms/kg once daily, to be taken after a meal at the same time each day, dose based on corrected age for preterm infants.
- **Child 2-17 years (body-weight up to 20 kg)** (By mouth): 250 micrograms/kg once daily, to be taken after a meal at the same time each day.
- **Child 2-17 years (body-weight 20 kg and above)** (By mouth): 5 mg once daily, to be taken after a meal at the same time each day.
Side effects
Common or very common Arthralgia; cystitis; diarrhoea; fever; headache; hyperpyrexia; increased risk of infection; nausea; oral ulceration; skin reactions Frequency not known Cutaneous vasculitis
Interactions
**Severe interactions:**
- In vitro studies have shown that risdiplam and its major human metabolite M1 are not significant inhibitors of human MDR1, organic anion-transporting polypeptide (OATP)1B1, OATP1B3, organic anion...
**Other interactions (9):**
- Risdiplam is primarily metabolized by hepatic enzymes flavin monooxygenase 1 and 3 (FMO1 and 3), and also by cytochrome P450 enzymes (CYPs) 1A1, 2J2, 3A4, and 3A7.
- Risdiplam is not a substrate of human multidrug resistance protein 1 (MDR1).
- Effects of other medicinal products on risdiplam Co-administration of 200 mg itraconazole twice daily, a strong CYP3A inhibitor, with a single oral dose of 6 mg risdiplam did not exhibit a...
- Effects of risdiplam on other medicinal products Risdiplam is a weak inhibitor of CYP3A.
- In healthy adult subjects, oral administration of risdiplam once daily for 2 weeks slightly increased the exposure of midazolam, a sensitive CYP3A substrate (11% increase in AUC; 16% increase in C...
- However, risdiplam and its metabolite are in vitro inhibitors of the human organic cation transporter 2 (OCT2) and the multidrug and toxin extrusion (MATE)1 and MATE2-K transporters.
Pregnancy
Avoid-toxicity in animal studies. M
Breast feeding
Avoid-present in milk in animal studies. M
Medicinal forms
Clinical governance
**Author:** MedNext Clinical Team. **Clinical reviewer:** Dr Shameer Deen, MBBS, MS, MRCS. **Sources:** BNF, Indian Pharmacopoeia, CIMS India. **Correction:** Report errors at support@mednext.academy.
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