Indications
Risdiplam is used to treat spinal muscular atrophy (SMA), an inherited disease in which the nerve cells that control muscles are lost, causing muscle weakness and wasting. It is used in babies, children and adults with SMA types 1, 2 or 3, or with one to four copies of the SMN2 gene, as decided by a specialist.
How It Works
People with SMA lack a working SMN1 gene and depend on a back-up gene, SMN2, which makes only small amounts of the survival motor neuron (SMN) protein. Risdiplam changes the way SMN2 is read (splicing) so that the body makes more complete, working SMN protein. This helps protect the motor nerve cells and muscle function.
Dosage and Administration
Risdiplam is a specialist medicine taken by mouth once a day, after a meal (or after breastfeeding in babies), at about the same time each day. The dose depends on age and body weight and is given exactly as prescribed by the neurologist. The liquid is prepared by the pharmacist and measured with the oral syringe provided; it can also be given through a feeding tube. Tablets, where available, are taken as directed. Rinse with water after a dose and do not mix with milk or formula.
Interactions
Risdiplam may increase blood levels of medicines that are removed by MATE kidney transporters, such as Metformin; the doctor may adjust or monitor these. Tell the doctor about all medicines, vitamins and herbal products.
Contraindications
- Allergy to Risdiplam or any ingredient
Side Effects
Common effects include fever, diarrhoea, rash, mouth ulcers, joint pain, headache, nausea and urinary tract infection. In babies, upper respiratory infections and vomiting may occur.
Get urgent medical help for signs of a severe allergic reaction, a widespread or blistering rash, high fever that does not settle, breathing difficulty, or signs of dehydration in a baby (very few wet nappies, drowsiness).
Pregnancy and Lactation
Risdiplam may harm an unborn baby. Women who could become pregnant should have a pregnancy test before starting and use effective contraception during treatment and for at least 1 month after the last dose. It may affect male fertility; men should discuss sperm preservation before treatment and use contraception during treatment and for 4 months after the last dose. Breastfeeding is not recommended during treatment.
Precautions and Warnings
Use the oral syringe supplied for accurate dosing. Avoid getting the liquid on skin or in the eyes; wash with water if it happens. Keep regular follow-up with the SMA team. Do not stop treatment without advice.
Overdose
If too much is given, contact the doctor or hospital at once; there is no specific antidote and monitoring and supportive care are given.
Therapeutic Class
SMN2 splicing modifier; medicine for spinal muscular atrophy.
Storage
Before preparation, store the powder below 25°C. Once prepared by the pharmacist, store the oral solution in a refrigerator at 2 to 8°C, do not freeze, keep upright in the original amber bottle away from light, and use within the time written on the label. Tablets are stored as directed on the pack. Keep out of the reach of children.
Prepared by the Oshud Alo team from standard drug references. For information only; always follow your doctor's advice.