Friday, Jun 7, 2024

Five-Year Data for Genentech’s Evrysdi Show the Majority of Treated Children With a Severe Form of Spinal Muscular Atrophy (SMA) Achieved or Maintained the Ability to Sit, Stand or Walk

After 5 years of treatment, 91% of children were alive — without treatment, children with Type 1 SMA would not be expected to live past 2 years of age 

96% of Evrysdi-treated children could swallow, 80% could feed without a feeding tube and 59% could sit without support for at least 30 seconds 

Evrysdi is now approved in more than 100 countries with over 15,000 patients treated globally 

South San Francisco, CA -- June 7, 2024 --

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new 5-year data confirming the sustained efficacy and safety profile of Evrysdi® (risdiplam) in children with Type 1 spinal muscular atrophy (SMA) from the open-label extension of the pivotal FIREFISH study. By the end of year 5, 91% of children treated with Evrysdi were alive, 81% were alive without permanent ventilation and the majority were able to sit without support for at least 30 seconds (59%). At the end of year 5, seven children were able to stand, three with support, four unaided and six could walk with support. Without disease modifying treatment, natural history studies indicate that children with Type 1 SMA would not only never be able to reach such milestones, but also not typically live past the age of two. The data were presented at the Cure SMA Research & Clinical Care Meeting, June 5 - 7, 2024. 


“These long-term findings confirm the ongoing benefit of Evrysdi for children with Type 1 SMA,” said Professor Giovanni Baranello, M.D., Ph.D., UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, U.K. “Children treated with Evrysdi over 5 years have maintained or improved their ability to sit, stand and walk — critical skills for development and daily living. An overwhelming majority also maintained the ability to swallow and to eat without a feeding tube.” 


Motor function abilities, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) and Hammersmith Infant Neurological Examination 2 (HINE-2), were maintained or continued to be achieved in those treated with Evrysdi. The FIREFISH results showed most children treated with Evrysdi also maintained their feeding and swallowing abilities. Of those assessed at year 5, 96% were able to swallow and 80% were able to feed without a feeding tube. 


“This is the final readout of the FIREFISH study, which has provided a wealth of insights and data, helping to firmly establish Evrysdi as an important treatment option, improving the lives of children across the globe living with SMA,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “This would not have been possible without the commitment and dedication of the children and families who participated, as well as numerous healthcare professionals and patient support organizations to whom we are immensely thankful.”


No treatment-related adverse events (AEs) led to treatment discontinuation or withdrawal from the study. The overall rate of AEs decreased by 66% between year 1 and the final year of study. The most common AEs were upper respiratory tract infection (64%), pyrexia (64%) and pneumonia (50%). Hospitalizations declined over the 5-year treatment period and 22% of children did not require hospitalization at all since beginning treatment with Evrysdi.


Evrysdi is the only oral, non-invasive small molecule SMA treatment designed to be systemically delivered to both the central nervous system (CNS) and peripheral tissues. 


Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.


About Evrysdi® (risdiplam)

Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form either by feeding tube or by mouth.


Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues, as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core motor functions such as swallowing, speaking and breathing.


Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research Award for Drug Discovery. Evrysdi is currently approved in more than 100 countries, and the dossier is under review in a further 13 countries.


Evrysdi is currently being, or has been, evaluated in five global multicenter trials in people with SMA:

  • FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study. The study met its primary endpoint.
  • SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. The study met its primary endpoint.
  • JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174).
  • RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in babies (n=26), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint.
  • MANATEE (NCT05115110) – a Phase II/III clinical study to evaluate the safety and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is currently recruiting.


About SMA

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and is among the leading genetic causes of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.


What is Evrysdi?

Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.


Important Safety Information

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
    • are a woman who can become pregnant:
      • Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
      • 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
      • Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting 
    • are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine
    • are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
  • Tell your healthcare provider about all the medicines you take
  • You should receive Evrysdi from the pharmacy as a liquid. 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, and sore throat (upper respiratory infection)
      • lung infection (lower respiratory infection)
      • constipation
      • vomiting
      • cough


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 You may also report side effects to Genentech at 1-888-835-2555.


Please see full Prescribing Information for additional Important Safety Information.


About Genentech in Neuroscience

Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.


Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit


# # #