risdiplam at a glance
Medicine class
SMN2 pre-mRNA splicing modifier
Licensed role
Treatment of eligible people with 5q spinal muscular atrophy
Available forms
Film-coated tablets and a pharmacist-prepared oral solution
Food
May be taken with or without food
Feeding tubes
The oral solution can be given through a nasogastric or gastrostomy tube; tablets and tablet mixture cannot
What is risdiplam used for?
Risdiplam is licensed for 5q SMA in people with a clinical diagnosis of SMA type 1, 2 or 3, or with one to four SMN2 gene copies. This includes some people diagnosed genetically before symptoms develop.
Symptomatic 5q SMA type 1
Symptomatic 5q SMA type 2
Symptomatic 5q SMA type 3
Eligible genetically confirmed 5q SMA before symptoms develop
How does risdiplam work?
In 5q SMA, altered SMN1 genes cause a shortage of functional survival motor neuron protein. Risdiplam changes SMN2 pre-mRNA splicing, increasing production of stable, functional SMN protein in the central nervous system and elsewhere in the body.
risdiplam preparations and strengths
Oral solution
Route: Oral
Strengths: 0.75 mg/mL
Tablet
Route: Oral
Strengths: 5 mg
How to take risdiplam
Follow the specialist team’s instructions for the prescribed formulation.
Swallow tablets whole, or disperse them only in a small amount of room-temperature water. Do not chew, cut or crush them.
Take a dispersed tablet immediately. Do not use liquids other than water or expose the mixture to sunlight.
The oral solution is prepared by a healthcare professional. Measure it using the supplied reusable oral syringe and do not mix it with milk or formula milk.
Drink water afterwards where swallowing permits. Wash skin with soap and water after accidental contact.
Use the oral solution, not a tablet mixture, if administration through a nasogastric or gastrostomy tube is required.
risdiplam side effects
Common or expected effects
- Diarrhoea
- Rash
- Fever
- Headache
- Nausea
- Mouth ulcers
- Urinary tract infection
- Joint pain
Get urgent medical advice
- Possible cutaneous vasculitis, which may appear as reddish-purple or bruise-like spots and requires prompt clinical assessment
risdiplam in pregnancy
Human pregnancy data are unavailable, and animal studies found embryo-fetal harm. Risdiplam is not recommended during pregnancy. Pregnancy status should be checked before treatment, and highly effective contraception is required during treatment and afterwards. Contact the specialist promptly if pregnancy occurs or is planned.
risdiplam while breastfeeding
It is unknown whether risdiplam enters human breast milk, and potential harm to a breastfed child cannot be excluded. Breastfeeding is not recommended during treatment; discuss feeding options with the specialist team.
risdiplam interactions
Give the specialist and pharmacist a complete list of prescribed, over-the-counter and complementary products.
Metformin and other MATE1 or MATE2-K substrates
Risdiplam may increase blood concentrations; monitoring for related toxicity and review of the interacting treatment may be needed.
Nusinersen or other SMA-modifying treatments
Safety and efficacy of simultaneous use have not been established; specialist review is required.
Common questions about risdiplam
Answers are fully visible for fast scanning and source review.
Why is risdiplam used for 5q spinal muscular atrophy?
It targets SMN2 splicing, which can partly compensate for the faulty SMN1 gene responsible for 5q SMA. It is not established for unrelated causes of motor-neuron weakness.
Does risdiplam cure spinal muscular atrophy?
No. It increases functional SMN protein but does not correct the underlying genetic change or reverse all existing damage.
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.