Dear Members of the SMA Community,
We continue to work with the healthcare community to help people and families living with SMA get the support they need. As such, we wanted to share Biogen’s latest efforts on the following:
- We are committed to sharing information that will aid healthcare providers and individuals with SMA in making informed treatment decisions, and to providing new information and services for the SMA community in response to the COVID-19 pandemic.
- Biogen remains dedicated to advancing research in SMA to address the needs of the community and provide additional data to inform treatment decisions. Read further for information about a new study we plan to initiate.
We also want to congratulate Cure SMA for hosting a very successful first-ever virtual Annual SMA Conference and SMA Research & Clinical Care Meeting. Biogen presented key research updates from the SPINRAZA® (nusinersen) clinical development program and hosted a virtual symposium for researchers and providers focused on clinical and real-world experience with SPINRAZA in adults with SMA. We sponsored an online educational session for the SMA community featuring a panel of multidisciplinary care team members discussing their roles in treating SMA – and how COVID-19 has sparked new ways to engage virtually – and were proud to support virtual social activities specifically geared toward adults with SMA.
With SMA Awareness Month winding down, we would like to emphasize Biogen’s long-term dedication to this community. In recognition of the community’s great progress, Biogen hosted a variety of SMA Awareness Month activities, including challenging Biogen employees to participate in a virtual “race” from our Biogen headquarters to the Cure SMA headquarters. Our team is still logging their own walks and runs to travel the 993 miles in distance, while raising money for Cure SMA along the way. We will continue to keep the community posted on these efforts.
More information on our efforts can be found below. We look forward to sharing additional updates with you later this year.
Best regards,
The Biogen SMA Team
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Biogen Care Packages
In June, Biogen began distributing Patient Care Packages to SPINRAZA patients and their families in response to concerns related to COVID-19. These care packages include masks, gloves, hand sanitizer, and disinfecting wipes, which can be used if consistent with the advice of one’s healthcare team.
Biogen Virtual Education Programs
Additionally, Biogen will now host its educational programs for individuals living with SMA through a virtual platform. Throughout the summer and into the fall, we are offering live webinars which will cover a range of topics, including Navigating Health Insurance, Physical Assessments, and Multidisciplinary Care. More information on these programs can be found on the newly launched SPINRAZA website.
RESPOND Trial
As part of our ongoing commitment to the community, we continue to seek opportunities to build on our groundbreaking research to pursue improved outcomes for patients living with SMA.
Biogen is excited to announce that we plan to initiate a new global Phase 4 clinical study, RESPOND, to examine the clinical benefit and assess the safety of SPINRAZA in infants and children with SMA who still have unmet clinical needs following treatment with gene therapy.
Based on the planned study design, RESPOND will be a two-year, open-label study to evaluate the efficacy and safety of SPINRAZA in SMA patients previously treated with gene therapy to further inform treatment decisions. Efficacy will be assessed by change from baseline on motor function measures, additional clinical outcomes (e.g., swallowing), and caregiver burden. As a result of our ongoing discussions with the SMA community, we expect that data from the RESPOND study will help inform treatment decisions for our youngest SMA patients.
The study is projected to enroll 60 children up to 3 years old who are determined by the investigator to have the potential for additional clinical improvement after receiving gene therapy. The primary study group aims to include 40 infants aged 9 months or younger (at the time of first SPINRAZA dose) who have 2 copies of SMN2 (likely to develop SMA Type 1) and received gene therapy at 6 months old or younger. A second study group will include 20 children and will generate data in patients with a broader age range (up to 3 years old at the time of first SPINRAZA dose).
Biogen plans to submit the study protocol to the regulatory authorities in the coming months and aims for the first eligible patients to be enrolled in the RESPOND study in Q1 2021. More details about the RESPOND study are available here.
We remain committed to addressing the needs of the SMA community and look forward to your continued partnership.
INDICATION
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.