Evrysdi helped infants achieve a key motor milestone and survive without permanent breathing support

Some infants achieved the ability to sit without support

Sitting

Evrysdi helped infants achieve a key motor milestone

Out of the infants taking the recommended dose of Evrysdi for 12 months 

41% of infants sat without support for at least 5 seconds. 7 out of 17 infants.
Untreated infants with Type 1 SMA are not expected to sit without support

The infants in the study ranged in age from 3 to 7 months at the start of treatment, but many were closer to 7 months old.

Exploratory observations: results after 24 months

AFTER 24 MONTHS

59% of infants (10/17) taking the recommended dose of Evrysdi sat without support for at least 5 seconds as measured by BSID-III.

This information is considered exploratory. This means it was not designed to show a treatment effect so conclusions cannot be drawn.

Survival

Evrysdi helped infants survive without permanent breathing support

Out of the infants taking Evrysdi

At 12 months, 90% were alive and could breathe without permanent support (19 out of 21 infants). At 23 months, 81% were alive and could breathe without permanent support (17 out of 21 infants)

The infants in this part of the study ranged in age from 3 to 7 months at the start of treatment, but many were closer to 7 months. 

Additional exploratory observations in eating and swallowing

This information is considered exploratory. This means it was not designed to show a treatment effect so conclusions cannot be drawn.

Out of the infants taking the recommended dose of Evrysdi for 12 months:

  • 88% were able to eat by mouth (15 out of 17 infants)
  • 88% were able to swallow (15 out of 17 infants)

*Permanent support was defined as having a tracheostomy (a surgery where a tube is inserted in the front of the throat into the windpipe) or more than 21 days of noninvasive ventilation support (16 or more hours a day) or being intubated (a procedure where a breathing tube is inserted down the throat and into the windpipe) to help with breathing in the absence of an acute reversible event.
Two of these infants could eat some food by mouth, but also used a feeding tube.
One infant could not swallow at the start of the study.

Measurement tool

More about BSID-III

The results shown were measured using a tool called The Bayley Scales of Infant and Toddler Development Gross Motor Scale–Third Edition (BSID-III).

Who: Infants. How they're measured: Assessed for head control and their performance on activities such as rolling from side to side, sitting upright, and crawling motions
Talking with your healthcare provider

Use these questions to guide your discussion about Evrysdi with your healthcare provider

What is Evrysdi?

Evrysdi® (risdiplam) is a prescription medicine used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.

 

It is not known if Evrysdi is safe and effective in children under 2 months of age.

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • 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.