Evrysdi® (risdiplam) data further confirm long-term efficacy and safety data in a broad range of people with spinal muscular atrophy (SMA)

Latest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to sit, stand and walk within timeframes typical of healthy babies

New gene therapy data in boys living with Duchenne muscular dystrophy (DMD) reports encouraging results from early stage study supporting its progress into global pivotal Phase III EMBARK study

Basel, 08 March 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its growing neuromuscular portfolio will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, 13 – 16 March 2022. Presentations include eight abstracts from its spinal muscular atrophy (SMA) programme and three from its Duchenne muscular dystrophy (DMD) programme, demonstrating Roche’s commitment to advancing clinical understanding of these conditions with the aim of treating a broad range of people living with neuromuscular disorders.

“These new data further highlight the compelling body of evidence for Evrysdi across infants, children and adults living with SMA,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “The results from our Duchenne Muscular Dystrophy gene therapy programme are encouraging and represent the potential to provide transformative outcomes for people living with this condition. We are grateful for the partnerships that can help us accelerate progress across SMA and DMD.”

Spinal Muscular Atrophy (SMA)

Six abstracts from the Evrysdi® (risdiplam) clinical development programme will be presented. This includes 3-year SUNFISH Part 1 and 2 efficacy and safety data, as well as a comparison of 2-year SUNFISH Part 2 data with an untreated external control group, both of which highlight the long-term efficacy and safety profile of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA. The data also demonstrate sustained increase in motor function over time, compared to an untreated external control group at 2-years.

Updated interim data from the RAINBOWFISH study will report updated safety data in enrolled infants and efficacy data in infants who have received risdiplam for at least 12 months.

Roche will also be presenting data from four other SMA studies including:

24-month data from FIREFISH Part 1 and 2 in SMA Type 1, exploring the safety and efficacy of Evrysdi in infants treated with Evrysdi for 2-years

12-month data from the JEWELFISH study, exploring the safety, tolerability and pharmacokinetic/pharmacodynamic (PD) relationship of Evrysdi in a broad age range of patients (6 months to 60 years) who have previously received RG7800 (RO6885247), nursinersen, olesoxime or onasemnogene abeparvovec

Design of new MANATEE trial, a multi-centre, randomised, placebo-controlled, double-blind study investigating the effect of GYM329, an investigational anti-myostatin, in combination with Evrysdi

Data on the demographics and clinical characteristics of people with SMA enrolled in the Muscular Dystrophy Association Neuromuscular Observational Research (MOVR) hub

Using baseline data from FIREFISH, SUNFISH, JEWELFISH and NAtHis-SMA studies, a validated algorithm for estimating the weight of patients with SMA based on few input parameters

Duchenne Muscular Dystrophy (DMD)

Roche has partnered with Sarepta to accelerate access to investigational gene therapy, delandistrogene moxeparvovec (SRP-9001), for DMD outside of the United States upon US Food and Drug Adminstration (FDA) approval. Sarepta, will present three year data from Study 101, an open-label Phase 1/2a study evaluating the safety of SRP-9001 in four ambulatory participants aged between 4-7 years old with DMD. The results show an acceptable safety profile, and all patients demonstrated a clinically meaningful improvement in their North Star Ambulatory Assessment (NSAA) from baseline. In addition, Sarepta will also present results from Study 102 which continue to support SRP-9001’s clinical profile and reinforce a potential benefit risk profile. Study 102 is a three-part, Phase 2 clinical study evaluating the safety and efficacy of delandistrogene moxeparvovec in patients with DMD. Analysis of Part 2 of the study will be presented.

Other presentations include:

The study design and methodology of the first delandistrogene moxeparvovec global Phase III gene therapy trial, EMBARK, assessing the safety and efficacy of commercially representative delandistrogene moxeparvovec material in ambulatory boys with a confirmed DMD mutation aged 4 to 7 years old.

The full range of data from Roche’s clinical development programme in neuroscience being presented at MDA 2022 include:



Full session details and data presentations listing for the MDA Clinical and Scientific Conference can be found at the meeting website: https://mdaconference.org/

Follow Roche on Twitter via @Roche and keep up to date with MDA 2022 Conference news and updates by using the hashtag #RocheAtMDA2022, #MDA and #neuromuscular

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 SMN protein deficiency. To date over 5,000 patients have been treated with Evrysdi, which is administered daily at home in liquid form by mouth or by feeding tube.

Evrysdi is designed to treat SMA by increasing and sustaining the production of the survival motor neuron (SMN) protein in the central nervous system (CNS) and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

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 75 countries and the dossier is under review in a further 27 countries.

About SMA

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause 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.

About delandistrogene moxeparvovec

Delandistrogene moxeparvovec (SRP-9001; rAAVrh74.MHCK7.micro-dystrophin) is an investigational gene therapy aimed to deliver the micro-dystrophin-encoding transgene directly to the skeletal and cardiac muscle for the targeted production of the micro-dystrophin protein to enable a durable clinical response. Sarepta Therapeutics is responsible for global development and manufacturing for delandistrogene moxeparvovec and plans to commercialize delandistrogene moxeparvovec in the United States upon receiving FDA approval. In December 2019, Roche partnered with Sarepta to combine Roche’s global reach, commercial presence, and regulatory expertise to accelerate access to delandistrogene moxeparvovec for patients outside the United States.

About DMD

DMD is a rare X-linked, progressive neuromuscular disease caused by mutations in the DMD gene that disrupts the production of functional dystrophin protein, leading to a loss of muscle function and premature death. DMD is one of the most common fatal genetic disorders, affecting approximately one in every 3,500 to 5,000 male births worldwide.

Symptoms usually appear in infants and toddlers, with affected children presenting developmental delays such as difficulty walking, climbing stairs or standing from a sitting position. As DMD progresses, muscle weakness involves the arms, trunk, and other areas, meaning patients often require full-time use of a wheelchair in their early teens. Longevity is limited due to cardiac and/or respiratory failure.

About Roche in Neuroscience

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

Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, 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 Roche

Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan