Dear Members of the SMA Community,
Each year, the entire rare disease community comes together on the last day of February to recognize and raise awareness for the 300 million people worldwide, including 25-30 million Americans, impacted by a rare disease like SMA: that is one in 10 Americans, and more than half of them are children. Biogen was proud to sponsor Rare Disease Day activities worldwide, led by the National Organization for Rare Disorders (NORD) and Rare Diseases Europe (EURORDIS).
In the coming months, Biogen will support many virtual events and activities, which allow the SMA community to get involved, keep informed, and stay connected to each other and to the science. Meetings early on this year, like the Muscular Dystrophy Association’s (MDA) Annual Clinical and Scientific Conference and Cure SMA’s Annual SMA Conference are particularly important and we are proud to be able to sponsor both. Connecting with members of the SMA community at these and other events continues to be a highlight for us at Biogen.
We also continue to invest in new research and development programs for our SMA therapy, SPINRAZA (nusinersen). This includes research into whether additional clinical benefit can be achieved with higher doses of nusinersen (DEVOTE study) or when SPINRAZA is given following gene therapy treatment (RESPOND study). We are excited to share that we enrolled the first patient into Part B of DEVOTE at the end of 2020 and the first patient into RESPOND just after the start to 2021.
The Biogen SMA Team
Rare Disease Day 2021
Biogen held several internal events and activities related to Rare Disease Day and was a proud sponsor of many external events as well, including those organized by NORD, EURORDIS, and MassBio (the Massachusetts Biotechnology Council). At Biogen, we work every day to pioneer new science that can help us better understand, and eventually treat, many rare diseases, including SMA. We are closely connected to these communities and have a deep appreciation for the advocates, organizations, patients, caregivers, and healthcare providers fighting on the frontlines for the needs of those with SMA and other rare diseases.
We were also honored to participate in an event hosted by the Reuters news agency about unlocking innovation and access to medicines for rare disease patients in Europe. The event featured remarks from rare disease advocates, as well as political and healthcare leaders, including Ursula von der Leyden, President of the European Commission, Biogen’s Johanna Friedl-Naderer, President of Europe, Canada & Partner Markets, among others.
Continuing to Support the SMA Community Through Virtual Activities and Events
We are proud to sponsor Cure SMA’s online book club, a series of trivia nights, virtual socials for adults with SMA, two seminars on at-home physical therapies techniques, and three seminars exploring topics related to career development.
Biogen also continues to offer educational live and virtual events for people with SMA and their families. Learn more about SPINRAZA and get firsthand information from other members of the SMA community. Upcoming Support and Treatment Education Programming (STEP) from Biogen includes session topics such as Advocating for Optimal SMA Care, What to Expect When Transitioning SMA Care, Understanding Intrathecal Administration, Assessing Motor Function in SMA. Information contained within STEP programs is intended only for U.S. audiences and is not intended to replace discussions with healthcare providers. For more information about each session, including roster of speakers click here.
Additional Updates on Biogen’s Clinical Trial Program
RESPOND Study Kicks Off
Biogen announced at the start of 2021 that the first person was treated with SPINRAZA in the RESPOND study, a global Phase 4 study examining the clinical benefit and assessing the safety of SPINRAZA in infants and children who have unmet clinical needs following treatment with Zolgensma (onasemnogene abeparvovec-xioi). More information about the RESPOND trial can be found here.
DEVOTE Study Advances Forward
Biogen’s DEVOTE study is designed to evaluate the safety, tolerability, and efficacy of nusinersen when administered at a higher dose than currently approved for the treatment of SMA. While all participants in this study receive nusinersen, some of those in Part B will receive a higher dose of nusinersen.
The first participant in Part B was dosed in November 2020. Since then, several additional sites have been activated in the US. Check out the study website (www.devotesmastudy.com) for more information.
SPINRAZA® (nusinersen) is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.
IMPORTANT SAFETY INFORMATION
Increased risk of bleeding complications has been observed after administration of some similar medicines. Your healthcare provider should perform blood tests before you start treatment with SPINRAZA and before each dose to monitor for signs of these risks. Seek medical attention if unexpected bleeding occurs.
Increased risk of kidney damage, including potentially fatal acute inflammation of the kidney, has been observed after administration of similar medicines. Your healthcare provider should perform urine testing before you start treatment with SPINRAZA and before each dose to monitor for signs of this risk.
The most common possible side effects of SPINRAZA include lower respiratory infection, fever, constipation, headache, vomiting, back pain, and post-lumbar puncture syndrome.
These are not all the possible side effects of SPINRAZA. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Before taking SPINRAZA, tell your healthcare provider if you are pregnant or plan to become pregnant.
Please see full Prescribing Information.
This information is not intended to replace discussions with your healthcare provider.
- Nguengang Wakap, S., Lambert, D.M., Olry, A. et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database. Eur J Hum Genet 28, 165–173 (2020). https://doi.org/10.1038/s41431-019-0508-0
- Global Genes. Rare Facts. Available at: https://globalgenes.org/rare-facts/. Accessed: February 2021.