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FDA Approves Genentech’s Treatment for Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older

August 7th, 2020

On August 7, 2020, the FDA approved Genentech’s Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Evrysdi showed clinically-meaningful improvements in motor function across two clinical trials in people with varying ages and levels of disease severity, including Types 1, 2, and 3 SMA. A liquid medicine, Evrysdi is administered daily at home by mouth or feeding tube.


News in brief

  • In two clinical trials, Evrysdi improved motor function in people living with SMA over a broad spectrum of ages and levels of disease severity, including Types 1, 2, and 3 SMA
  • Evrysdi is the first and only medicine for SMA that can be taken at home
  • SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality.

Supporting Information

Important Safety Information

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • have liver problems
    • are pregnant or plan to become pregnant. If you are pregnant, or are planning to become pregnant, ask your healthcare provider for advice before taking this medicine. Evrysdi may harm your unborn baby.
    • are a woman who can become pregnant:
      • Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy. Because Evrysdi may harm your unborn baby, your healthcare provider will decide if taking Evrysdi is right for you during this time
      • Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi
    • are an adult male planning to have children: Evrysdi may affect a man’s ability to have children (fertility). If this is of concern to you, make sure to ask a healthcare provider for advice
    • are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby. If you plan to breastfeed, discuss with your healthcare provider about the best way to feed your baby while on treatment with Evrysdi
  • Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine
  • You should receive Evrysdi from the pharmacy as a liquid that can be given by mouth or through a feeding tube. The liquid solution is prepared by your pharmacist. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement
  • Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water
  • The most common side effects of Evrysdi include:
    • For later-onset SMA:
      • fever
      • diarrhea
      • rash
    • For infantile-onset SMA:
      • fever
      • diarrhea
      • rash
      • runny nose, sneezing, sore throat, and cough (upper respiratory infection)
      • lung infection
      • constipation
      • vomiting

    These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.

    You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

    Please see full Prescribing Information for additional Important Safety Information.


Levi Garraway, M.D., Ph.D.

“Given the majority of people with SMA in the U.S. remain untreated, we believe Evrysdi, with its favorable clinical profile and oral administration, may offer meaningful benefits for many living with this rare neurological disease.”