SMA Newsroom

Clinical trial data updates

Throughout 2021, Roche presented findings from the four ongoing clinical trials of risdiplam in people living with SMA. The growing body of data is essential to continue monitoring the longer-term safety and efficacy profile of risdiplam and to furthering collective scientific knowledge.

SUNFISH

(Evaluating risdiplam in people aged 2-25 years with Type 2 or non-ambulant Type 3 SMA)

During the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical and Scientific Conference, Roche presented 24-month efficacy and safety data from Part 2 of the SUNFISH study. For a more in-depth look at the findings, please access the press release here

FIREFISH

(Evaluating risdiplam in infants aged 1-7 months at enrolment with symptomatic Type 1 SMA.)

At the 73rd American Academy of Neurology (AAN) Annual Meeting, Roche presented 24-month efficacy and safety data from Part 2 of the FIREFISH study. For a more in-depth look at the findings, please access the press release here

JEWELFISH

(Evaluating risdiplam in people with all types of SMA aged 1 to 60 years previously treated with another SMA-targeting therapy, including nusinersen and onasemnogene abeparvovec)

Safety, pharmacodynamic and exploratory efficacy data from the JEWELFISH study were presented during the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting. More information on the data presented can be accessed in the press release here

RAINBOWFISH

(Evaluating the efficacy and safety of risdiplam in babies from birth to six weeks with pre-symptomatic SMA)

Preliminary efficacy and safety data from the RAINBOWFISH study were shared during the Cure SMA 2021 Virtual SMA Research & Clinical Care. For a more in-depth look at the findings, please access the press release here

The data presented further demonstrated a favourable efficacy and safety profile for risdiplam. Our next update will be shared during the SMA Europe Scientific and Clinical Congress 2022 in February, which we are proud to support.

New research and development programmes

Roche continues to invest in new research and development programmes, in pursuit of new treatment pathways that can address the ongoing needs in the SMA community. One approach under evaluation is combining treatments that work in different ways, which is believed to have the potential to provide further benefit to people impacted by SMA, by addressing the underlying cause and the symptoms of the disease concurrently.[1]

In October, Roche announced the initiation of MANATEE, a two-part, global Phase 2/3 clinical study, which aims to evaluate the safety and efficacy of GYM329 (RO7204239), an investigational anti-myostatin antibody targeting muscle growth in combination with risdiplam. Approximately 15 sites have been selected to participate in Part 1 of MANATEE, which is anticipated to start in early 2022. Whilst the study is currently enrolling ambulant patients aged 2-10 years, they will also be exploring the combination in other patient populations in the future, including non-ambulant patients and in a broader age range. More information regarding the study and its trial sites can be found on ClinicalTrials.gov.

Access to risdiplam

Since the first regulatory approval in the US in August 2020, risdiplam has been approved in 66 countries worldwide [2], including all 27 European Union member states as well as Iceland, Norway and Liechtenstein. Roche is continuing to pursue regulatory approvals. Applications are currently under review in 34 additional countries [3], reaching a total of 100 submissions and approvals. To date, more than 4,500 people have been treated with risdiplam in clinical trials, or have received risdiplam commercially and through the compassionate use programme, which is open in more than 60 countries.

Regulatory approval is only the first step towards patient access. Roche is working closely with reimbursement and assessment bodies in European countries and worldwide to minimise any delay between regulatory approval and access to risdiplam and ensure sustainable access. To date, 13 countries (Australia, Denmark, France, Germany, Iraq, Japan, Kuwait, North Macedonia, Qatar, Russia, United States, Serbia, Switzerland) have national reimbursement for the use of risdiplam and an additional 10 countries (Canada, Croatia, England, Estonia, Latvia, New Zealand, Northern Ireland, Sweden, Ukraine, Wales) have positive national assessment decisions (with reimbursement pending). The reimbursement and assessment decisions are specific to each country. Roche is looking forward to providing more details as part of their support of our OdySMA Project.

Roche’s ongoing commitment

2021 marked 10 years of Roche working in partnership with the SMA community.  Over this decade, the company has worked collaboratively with their partners in the community to make a difference to the lives of people living with SMA, their families and loved ones. During 2021, Roche worked on a number of collaborative projects through their engagement with us directly and the broader community.

As a company they are honoured to be part of two important collaboration initiatives, led by us, that aim to improve healthcare and reduce disparities. The first is the European Alliance for Newborn Screening in SMA, founded in 2020, which advocates for the inclusion of SMA in all newborn screening tests in Europe in order to expedite the time to a correct diagnosis, which is vital to preventing the onset of disability and in maintaining the best possible quality of life. Recently, Germany and Norway became the first two European countries to announce nationwide newborn screening for SMA and many other countries are taking bold steps towards achieving that goal. In addition, Roche is pleased to expand their partnership with us and support our OdySMA Project in our joint efforts to address the challenges of access to SMA treatments and care across Europe and ensure that no subgroup is left untreated.

At the same time, Roche has continued to invest in advancing collective knowledge on important topics including the adaptation of standards of care and building capabilities in Health Technology Assessment. By strengthening connectivity, exchanging best practices and learnings, they help further the work to address unsolved challenges and take a step towards delivering equal healthcare across the globe.

Finally, Roche feels privileged to have worked together with the community to launch SMA My Way, an online collaboration to support people impacted by SMA by providing a dedicated space for sharing personal experiences, or life-hacks and empowering one another to celebrate individuality and pursue life goals.

Roche is proud of the work undertaken collaboratively and the company remains committed to providing its ongoing support for these important efforts. They thank us for the trust we have placed in them and for our continuous partnership, which has been instrumental in shaping their clinical development programmes and delivering solutions that are centred around the community’s expectations and needs.

Finally, Roche wishes everyone in the SMA community a happy holiday season and looks forward to continuing this collaboration in 2022!

• Cure SMA. Scientific Considerations For Drug Combinations.
• Approved: US, EU (EU 27 + Norway + Iceland + Liechtenstein), Aruba, Australia, Azerbaijan, Brazil, Chile, China, Canada, Dominican Republic, Ecuador, Egypt, El Salvador, Georgia, Guatemala, Guyana, Honduras, Hong-Kong, India, Indonesia, Israel, Japan, Kuwait, Malaysia, North Macedonia, Paraguay, Peru, Qatar, Russia, Singapore, South-Korea, Switzerland, Thailand, Ukraine, UAE, UK, Uruguay.
• Submitted: Albania, Argentina, Belarus, Bolivia,  Bosnia-Herzegovina, Botswana, Colombia, Costa Rica, Cuba, Curacao, Jordan, Kazakhstan, Libya, Mauritius, Mexico, Moldova, Montenegro, Morocco, Namibia, Nicaragua, New Zealand, Oman, Panama, Pakistan, Philippines, Saudi Arabia, Serbia,  Sint-Maarten, South Africa, Trinidad & Tobago, Turkey, Taiwan, Vietnam, Zambia
• The community letter – 16th December 2021