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Dear SMA Community,

As 2020 draws to a close, we want to provide an overview of our recent work in the community over the fourth quarter and updates related to clinical studies of Evrysdi™ (risdiplam), which was approved earlier this year for the treatment of spinal muscular atrophy (SMA) in people 2 months of age and older.

Our Ongoing Commitment

While this year has been uniquely challenging for the population at large due to the COVID-19 global pandemic, we understand that circumstances are particularly difficult for those living with SMA. Our goal has been to meet the community where they are and have been pleased to participate in several virtual Summit of Strength programs throughout the fall. At these events, we were able to explain the role of our Patient and Access Liaisons (PALs) in providing families with information about Evrysdi, as well as guidance on receiving the medicine and assistance with navigating insurance and financial support, once a person has been prescribed Evrysdi. We were also honored to participate in Cure SMA’s Chapter Leadership Summit, where we had a chance to hear directly from chapter leaders about the challenges currently facing their communities, and learn about the work that needs to be done to better support those communities in the year ahead.

We understand that patients continue to face geographic barriers in reaching care, feel isolated due to pandemic-imposed shutdowns, and struggle to locate medical supplies due to the burdens placed on the healthcare system. As an initial step in the effort to address these needs, we were pleased to support a Cure SMA initiative that provided a second round of Cure SMA COVID-19 SMA Community Support Packages, along with a series of wellness webinars, virtual therapy options, and morning coffee education sessions. We are hopeful that these extended and new programs will provide valuable avenues for connection and support during these difficult times. As we turn the page to 2021, we look forward to continuing our support of all members of the SMA community.

Q4 Scientific Presentations and Study Updates

In October, at the World Muscle Society Congress, we presented data from our four “FISH” studies, including 2-year data from Part 1 of the pivotal FIREFISH study of Evrysdi in infants aged 3-7 months with symptomatic Type 1 SMA.

The continuation of FIREFISH, along with the SUNFISH, JEWELFISH, and RAINBOWFISH studies, will further our understanding of Evrysdi’s efficacy and safety, as well as evaluate safety and efficacy in additional populations. We look forward to sharing the results of these studies at future scientific conferences.

As a reminder, JEWELFISH is investigating the safety and tolerability of Evrysdi in people with all types of SMA aged 6 months to 60 years, who have been previously treated with other SMA therapies.

SUNFISH is a large (n=231) global, two-part, open-label study evaluating the efficacy and safety of Evrysdi in people with Type 2 or 3 SMA aged 2 to 25 years.

RAINBOWFISH is an open-label, single-arm, multicenter study (n=25) investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in infants from birth to 6 weeks old who have been genetically tested and diagnosed with SMA, but have not yet shown disease symptoms. This is the only global clinical trial with Evrysdi currently recruiting. More information about RAINBOWFISH and its trial sites can be found on the ClinicalTrials.gov website. To reiterate, Evrysdi is not approved for people with SMA that are younger than 2 months of age.

As always, we extend our sincere gratitude and appreciation to the many patients and families who are participating in our ongoing clinical studies. We continue to be humbled to serve this resilient community, in a new way, now that Evrysdi has been approved by the U.S. Food and Drug Administration (FDA), and are grateful for everything we have achieved by working together.

We are pleased to share that since August, seven additional countries (Chile, Brazil, India, Ukraine, South Korea, Georgia, and Russia) have also approved the use of risdiplam in the last few months. Furthermore, we anxiously await decisions from regulatory authorities on 21 existing regulatory applications under review in 47 countries worldwide, including the 27 member states of the European Union, Norway, Iceland, Indonesia, Taiwan, China, Australia, Canada, Israel, Malaysia, Switzerland, Thailand, UAE, Macedonia, Kuwait, Singapore, New Zealand, Japan, Peru, South Africa, and Pakistan.

We wish you a happy and healthy holiday season and look forward to the year ahead.

Sincerely,

Genentech SMA Team

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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.