Evrysdi™ (risdiplam)

Reviewed by: HU Medical Review Board | Last reviewed: May 2024 | Last updated: May 2024

In 2020, the US Food and Drug Administration (FDA) approved Evrysdi™ (risdiplam) to treat adults and children over 2 months old with spinal muscular atrophy (SMA). In 2022, that approval was extended for treating infants younger than 2 months old. Evrysdi works by increasing the ability of motor neurons to produce the “survival of motor neuron” (SMN) protein.1

In clinical trials, children and adults treated with Evrysdi showed improved motor function and survival rate. Ongoing clinical trials are testing if outcomes are better when started earlier.2-9

Evrysdi is the first and only treatment that can be taken by mouth and at home. It is also cheaper than other FDA-approved treatments. However, it needs to be taken each day, which may make it less convenient for some people.10,11

What are the ingredients in Evrysdi?

The active ingredient in Evrysdi is risdiplam.1

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How does Evrysdi work?

Risdiplam, the active ingredient in Evrysdi, improves the ability of motor neurons to produce the “survival of motor neuron” (SMN) protein. It does this by changing how the backup SMN2 gene is processed. This helps keep motor neurons alive and prevents muscle wasting.1,12,13

One way to imagine this is that the SMN2 gene is a recipe for your cells to make the SMN protein. But it has important instructions missing, which makes the recipe unreadable. Risdiplam makes the recipe readable by fixing how motor neurons process the SMN2 gene.

The other FDA-approved drugs for SMA are large biological molecules. Evrysdi is a much smaller drug. The small size allows it to access spinal motor neurons from the bloodstream. This is why it can be taken by mouth.12,14

What are the possible side effects?

The most common side effects in children or adults with type 2 or 3 SMA include:1

  • Fever
  • Diarrhea
  • Rash

Infants with type 1 SMA can experience the same side effects. Other common side effects in infants include:1

  • Upper respiratory tract infection (runny nose, sneezing, sore throat, and cough)
  • Lung infection, including pneumonia and bronchitis
  • Constipation
  • Vomiting

These are not all the possible side effects of Evrysdi. Talk to your doctor about what to expect when taking Evrysdi. You also should call your doctor if you have any changes that concern you when taking Evrysdi.

Things to know about Evrysdi

Evrysdi is taken by mouth once a day after a meal. If there are difficulties swallowing, Evrysdi can also be taken by feeding tube.1,10

There is not enough data to know if Evrysdi is safe to take when pregnant or breastfeeding. Before taking Evrysdi, talk to your doctor if you are pregnant, planning to become pregnant, or are breastfeeding.1

The annual cost of Evrysdi depends on the dosage but is less than Spinraza. The maximum price is $340,000 per year. For an infant under 15 pounds, the annual price will be less than $100,000. Even still, most people with SMA will rely on insurance coverage or payment assistance programs to afford Evrysdi.11

Evrysdi can cause problems when taken with certain other drugs, such as metformin. Before beginning treatment for SMA, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

For more information, read the full prescribing information of Evrysdi.

Treatment results and side effects can vary from person to person. This treatment information is not meant to replace professional medical advice. Talk to your doctor about what to expect before starting and while taking any treatment.