A new treatment option for the SMA community Sponsored Post

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by Joe Pierce |

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Treatment options for spinal muscular atrophy (SMA) have become available in recent years, as research about SMA itself continues to expand.

This summer, the SMA community received exciting news, as the FDA approved a new treatment option called Evrysdi (risdiplam). It’s a prescription medication used to treat SMA in infants, children, and adults, in people 2 months and older. It is not known if Evrysdi is safe and effective in children under 2 months of age.

How Evrysdi is designed to work

SMA is a genetic condition caused by the shortage of a key protein called SMN, or survival motor neuron, which is needed for nerves and muscles to function properly.

Evrysdi is designed to work by helping to make and maintain more SMN protein. In animal studies, Evrysdi was distributed throughout the body.

Who is participating in the clinical studies?

Evrysdi is being studied in over 450 people with Type 1, 2, or 3 SMA, from 2 months to 60 years old. Efficacy of Evrysdi was only studied in people from 2 months to 25 years old.

A closer look at results in infants with Type 1 SMA

FIREFISH is a 2-part, open-label study of 62 infants ages 2-7 months with Type 1 SMA. Part 1 explored the recommended dose of Evrysdi for 21 infants, as well as safety and effectiveness of Evrysdi after 12 months. At 12 months, the infants were measured on their ability to sit without support for at least 5 seconds, and survival without permanent breathing support.

These results were measured using a tool called the Bayley Scales of Infant and Toddler Development Gross Motor Scale-Third Edition (BSID-III), which is used to measure sitting (item 22) and other developmental movements.

Additionally, 90% (19 out of 21) of the infants taking Evrysdi survived without permanent breathing support at the 12-month evaluation point. At 23 months, 81% (17 out of 21) of the infants survived without permanent breathing support.

Important Safety Information

Before taking Evrysdi, tell your doctor if you have liver problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. Evrysdi may harm an unborn or breastfed baby. Evrysdi may affect a man’s ability to have children (fertility). Tell your doctor about all the medicines you take.

These are not all the possible side effects of Evrysdi. Please refer to additional Important Safety Information below.

Results in children and adults with Type 2 or 3 SMA

SUNFISH is 2-part, placebo-controlled study of 231 children and adults (2 to 25 years old) with Type 2 or 3 SMA and a range of motor functions. The children and adults participating in this study have varying symptoms and motor functions; some have severe scoliosis or joint contractures. The main measurement in part 2 of this study was the change in motor function in 180 participants, as evaluated by MFM-32 after 12 months of treatment,§ compared with those not taking Evrysdi.||

In Part 2, 115 children and adults were treated with Evrysdi, while 59 people were on placebo, a substance that has no active medication and is often used in studies. After 12 months, those taking Evrysdi showed more improved motor function. The average MFM-32 score increased 1.36 points for those taking Evrysdi, and decreased 0.19 points for those not taking Evrysdi.

The Motor Function Measure-32 items (MFM-32) scale evaluates 32 different elements to assess head and trunk motor movement and limb motor movements. This scale is designed to capture change for a broad range of people. The scale measures motor function abilities such as standing and transfer movements (an example of a transfer movement would be moving from sitting in a chair to standing up), upper and lower body movements, and hand and foot movements with a point system ranging from 0-3. For example, the inability to start the movement would be a 0 on the scale, and the ability to partially complete the exercise would be a 1.

A third, supportive study, JEWELFISH, is looking at the safety of Evrysdi in 174 people with Type 1, 2, or 3 SMA from 1 to 60 years old who were previously treated with other SMA medications.

The first and only at-home treatment for SMA

It’s a liquid treatment, taken by mouth or feeding tube once per day, and a 1-month supply is shipped directly to you by a specialty pharmacy.

Could Evrysdi be right for you?

If you’re considering treatment for SMA, it may be the right time to talk with your healthcare provider about Evrysdi.

You can learn more about Evyrsdi and available support services at www.Evrysdi.com.

MySMA Support is here for you

MySMA Support is a support service from Genentech that can provide information about Evrysdi, including product education, insurance and financial assistance, coordination with the specialty pharmacy for medication delivery, and more.

The MySMA Support team includes Partnership and Access Liaisons (PALs), who are the local, main points of contact from Genentech.

Find your local PAL and learn how MySMA Support could help.

The MySMA Support team, including the PAL, does not provide medical advice and is not a substitute for your medical team. As always, your healthcare provider should be your main resource for any questions about your health and medical care.

*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.

Measured by Item 22 of the BSID-III Gross Motor Scale.

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.

§Measured by the MFM-32 scale.

||Those not taking Evrysdi were on placebo, a substance that has no active medicine and is often used in studies.

In some studies, including this one, if someone’s data cannot be collected on time for any reason, their progress cannot be counted in that part of the study. This chart only includes the information that was collected on time.

What is Evrysdi?

Evrysdi 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.

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.

FDA stands for the US Food and Drug Administration.

© 2020 Genentech USA, Inc. All rights reserved.
M-US-00007407(v1.0)  10/2020