Roche presents new data at World Muscle Society (WMS) 2021


  • New data shows pre-symptomatic babies with spinal muscular atrophy (SMA) treated with Evrysdi retained the ability to swallow
  • Evrysdi has demonstrated consistent clinically significant efficacy in adults, children and infants two months and older and is now approved in 58 countries around the world
  • Other presentations included data from studies supporting the efficacy, safety and durability of gene therapy, SRP-9001, in the treatment of Duchenne muscular dystrophy (DMD)

Basel, September 24, 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced data from its entire growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress of September 20-24, 2021. The presentations included additional results from the RAINBOWFISH Study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to birth. Six week age and data supporting further clinical investigation of gene therapy, SRP-9001, in Duchenne muscular dystrophy (DMD).

“These new data for Evrysdi may help extend the potential benefits of this drug to younger patients with ADS. Additionally, the data from SRP-9001 helped optimize the design of the next Phase III trial for DMD, ”said Levi Garraway, MD, Ph.D, Roche medical director and head of global product development. “Our goal is to continue to lead the way in the development of transformative drugs for neuromuscular diseases. We are grateful for the partnerships that help us develop new therapies for those affected by these devastating rare diseases. ”

At WMS 2021, data from the ongoing open study RAINBOWFISH was presented. Four in five of those treated with Evrysdi for at least 12 months have been able to stand and walk independently in the windows of the World Health Organization for healthy children. In addition, the five babies retained their ability to swallow and were able to eat exclusively orally after 12 months of treatment.

Previously reported results showed that infants treated with Evrysdi for at least 12 months achieved motor milestones in the Hammersmith Infant Neurologic Examination (HINE-2), with 100% (n = 5) able to maintain control of head, sit upright, roll and crawl.

These data add to the growing body of evidence supporting the efficacy of Evrysdi in a large patient population. Over 4000 patients have been treated with Evrysdi in clinical trials, compassionate use and real life situations.

In DMD, three-year data from the open-label trial, study SRP-9001-101, evaluating the safety of a single dose of experimental gene therapy SRP-9001 in four ambulatory children aged 4 and 7 years with DMD, were presented. The study showed that SRP-9001 was well tolerated with a key functional assessment, as measured by the North Star Ambulatory Assessment (NSAA), demonstrating an overall improvement in motor ability from baseline. The improvements in motor skills were maintained for three years, signifying a lasting response. The results support further clinical investigation to further assess the benefit / risk ratio of SRP-9001 in patients with DMD.

Roche and its partner Sarepta also shared data from ENDEAVOR (study SRP-9001-103), the first clinical trial using the commercially representative material SRP-9001 for the treatment of DMD. The interim results of the first 11 participants of cohort 1 (ambulatory boys aged 4 to 7 years) of the ongoing open phase 1b study, prove that SRP-9001 showed robust expression of micro-dystrophin and that no new safety signal has been identified.

In addition, the results of Part 1 of Study SRP-9001-102, an ongoing, randomized, double-blind, placebo-controlled clinical trial evaluating the safety, efficacy and tolerability of a single dose of SRP-9001 in 41 boys with DMD, showed that the study met its primary endpoint of change in micro-dystrophin protein expression from baseline. Participants treated with SRP-9001 generally showed an increase in the total NSAA score compared to placebo at 48 weeks, although this increase did not reach statistical significance compared to that in patients who received placebo. The safety profile was consistent with previous studies, and no new safety signals were identified.

The results of these studies provide important information for the ongoing clinical development program of SRP-9001, with the results of Studies 101 and 102 informing the design of the Phase 3 trial for SRP-9001, which is expected to begin in March. globally by the end of the year.

On Evrysdi® (risdiplam)
Evrysdi is a motor neuron survival splice modifier 2 (SMN2) designed to treat SMA by increasing the production of survival motor neuron protein (SMN). The SMN protein is found throughout the body and is essential for maintaining healthy motor neurons and movement. Evrysdi is administered daily at home as a liquid by mouth or through a feeding tube.

The United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older. Evrysdi achieved PRIME designation by the EMA in 2018 and orphan drug designation by the FDA and EMA in 2017 and 2019, respectively. To date, Evrysdi has been approved in over 50 countries, including the United States and Europe, and submitted in over 30 countries.

About SMA
AMS is a serious and progressive neuromuscular disease that can be fatal. It affects about one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene, which results in a deficiency of the SMN protein. This protein is found throughout the body and is essential for the functioning of the nerves that control muscles and movement.

Without it, nerve cells cannot function properly, resulting in muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and the ability to walk, eat, or breathe can be significantly reduced or lost.

About SRP-9001
SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) is an experimental gene therapy designed to deliver the gene encoding micro-dystrophin directly to skeletal and cardiac muscle for the targeted production of the protein micro-dystrophin to enable a clinical response sustainable. Sarepta Therapeutics is responsible for the global development and manufacturing of SRP-9001 and plans to market SRP-9001 in the United States after 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 SRP-9001 for patients outside of the United States.

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

Symptoms typically appear in infants and toddlers, with affected children with developmental delays such as difficulty walking, climbing stairs, or standing from a seated position. As DMD progresses, muscle weakness affects the arms, trunk, and other areas, which means patients often need a full-time wheelchair in their early teens. Longevity is limited due to heart and / or respiratory failure.

About Roche in Neuroscience
Neurosciences are 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 studying more than a dozen drugs for neurological disorders, including multiple sclerosis, neuromyelitis optic spectrum disorders, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease, Duchenne muscular dystrophy and autism spectrum disorders. 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 pharmaceutical and diagnostics pioneer focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics, along with growing capabilities in data-driven medical knowledge, help Roche deliver truly personalized healthcare. Roche works with partners in the healthcare industry to provide the best care for every person.

Roche is the world’s largest biotechnology company, with truly differentiated drugs in oncology, immunology, infectious diseases, ophthalmology and central nervous system diseases. Roche is also the world leader in in vitro diagnostics and tissue diagnostics for cancer, and a pioneer in the management of diabetes. In recent years, Roche has invested in genomic profiling and real-world data partnerships and has become a leading industry partner for medical knowledge.

Founded in 1896, Roche continues to research better ways to prevent, diagnose and treat disease and make a lasting contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty drugs developed by Roche are included in the World Health Organization’s Model Lists of Essential Medicines, including life-saving antibiotics, antimalarials and cancer drugs. In addition, for the twelfth year in a row, Roche has been recognized as one of the most sustainable companies in the pharmaceutical industry by the Dow Jones Sustainability Indices (DJSI).

The Roche group, headquartered in Basel, Switzerland, is active in more than 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and achieved sales of CHF 58.3 billion. Genentech, in the United States, is a full member of the Roche group. Roche is the majority shareholder of Chugai Pharmaceutical, in Japan. For more information, visit

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