Genentech Releases Evrysdi™ FDA Approval Letter to the SMA Community

Dear SMA Patient Advocacy Community,

As part of our ongoing partnership and following your request to receive updates about the risdiplam clinical development program, we are delighted to share with you a much-anticipated milestone. Today, the U.S. Food and Drug Administration (FDA) has approved risdiplam for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. Risdiplam will be known under its brand name Evrysdi™ (ev-RIZ-dee).

Today’s news means that risdiplam – a liquid medicine administered daily at home by mouth or feeding tube – will be available to people with SMA in the U.S.

This first health authority approval milestone for risdiplam has been an aspiration since our parent company, Roche, PTC Therapeutics and the SMA Foundation began collaborating 9 years ago. The strength and resolve of the SMA community have continually inspired us as we developed a new treatment option that has the potential to make a positive impact on patients’ lives. Today we celebrate our collective accomplishment.

Our sincere gratitude and appreciation go out to the many hundreds of patients and families who are participating in our clinical studies, as well as the many patient groups around the world. Thank you for your partnership, trust, and continued support that led to this important breakthrough. We are humbled to be part of this resilient community and grateful for everything we have achieved by working together.

Sincerely,

Alana Paull

Patient Advocacy Relations, Genentech

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
• 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.

© 2020 Genentech USA, Inc. All rights reserved. M-US-00006746(v1.0)