Biogen has released the following community statement on Spinraza access and new research data.

Dear Members of the SMA community,

Thank you for your continued support, efforts to raise awareness about SMA, and participation in clinical trials and studies as we seek to expand access to SPINRAZA® (nusinersen) for individuals with SMA around the world. We truly value working with you and remain your dedicated partner in increasing access to treatment for those who may benefit.

We are happy to report that more than 4,100 individuals with SMA are being treated with SPINRAZA worldwide.1 Due to your support, the SPINRAZA clinical development program continues to remain the largest and most robust clinical program for a disease-modifying therapy in SMA. We’d also like to share updates about our global access, new SPINRAZA research, advancements in treatment care in the US, and our investment in future treatment opportunities.

Global Access Update

SPINRAZA launches outside of the US continue at a rapid pace. Since the beginning of 2018, we:

  • Gained formal reimbursement in seven countries: Hungary, Slovenia, Israel2, Spain, Switzerland, Finland and Norway
  • Secured regulatory approval in Chile
  • Initiated named patient reimbursement in Portugal

Meanwhile, we are actively negotiating reimbursement in a number of countries, have several approvals pending, and are preparing filings in others – all with the goal of serving SMA communities around the world.

New Research Illustrating SPINRAZA’s Benefits for Pediatric and Adult SMA Patients

At the American Academy of Neurology (AAN) Annual Meeting last month and the Muscular Dystrophy Association (MDA) Clinical Conference in March, Biogen presented new research that underscores the benefits that SPINRAZA provides for pediatric and adult individuals living with SMA. Overall, the research showed SPINRAZA’s benefits continue over time across pre-symptomatic infants, infantile-onset, and later-onset SMA patients.

  • Long-term data for infantile-onset SMA: SHINE interim results from the open-label extension study, as of June 30, 2017, illustrated SPINRAZA’s longer-term benefits for 89 individuals with infantile-onset SMA, including improvements in motor function and increased chances of survival. Furthermore, SHINE participants experienced these benefits whether they initiated treatment earlier or at a later point in time. The research confirmed that those who initiated SPINRAZA treatment earlier saw greater motor milestone performance that continued to improve over time.
  • Data establishing impact of SPINRAZA on later-onset SMA: An analysis of clinical trial data showed that with SPINRAZA treatment, one individual with Type 2 and 13 individuals with Type 3 SMA – who were between two and 15 years old at study enrollment – walked longer distances while experiencing stable or decreased fatigue at the same time. It also illustrated that SPINRAZA’s benefits continue to grow over time for patients with later-onset SMA. Not only are these findings clinically meaningful, but they can translate to real-world benefits.
  • Treatment outcomes in pre-symptomatic infants: NURTURE interim results from the open-label study showed that all 25 pre-symptomatic infants treated with SPINRAZA were alive, did not require permanent ventaliation, and had improved motor function and motor milestone achievements as of July 5, 2017, compared to the disease’s natural history. The findings showcase the continued benefits that SPINRAZA provides for patients with SMA who started treatment in a pre-symptomatic state. Furthermore, 100 percent of NURTURE participants achieved the age-expected motor milestone of sitting without support, which is a development never seen with Type 1 SMA.
  • Growing data in young adults: A subset of five patients from the CS2/CS12 study who were 17 to 19 years old at the time of their last visit – four of whom had Type 3 SMA and one of whom had Type 2 – demonstrated that, with SPINRAZA, they had stable or slightly improved motor function and quality of life. This was seen during two years of treatment and even afterward, with improved and maintained stability and motor benefits.

Our Commitment to the US SMA Community & Advancing Treatment

We continue to build on broadening access to and reimbursement for SPINRAZA in the US, where we currently have more than 1,900 patients on therapy. Just over one year after SPINRAZA’s launch, a large number of infants have been treated and we are working to expand this progress among individuals with later-onset SMA. With adults constituting more than 50 percent of all SMA patients in the US, we are further encouraged by a recent increase in adult patients on therapy in Q1 2018.

In addition, it is encouraging to learn that HCPs are beginning to explore innovative approaches to access the intrathecal space in those individuals with complex spines, and have started to report this emerging experience in peer reviewed journals. Furthermore, we continue to work with clinical centers treating adults with SMA to understand barriers and evaluate potential solutions, and to ensure adequate support for adult patients interested in treatment.

Our Continued Focus on SPINRAZA and Future SMA Therapies

We remain dedicated to building a portfolio of treatments to support advancements in care for individuals with SMA. We are creating opportunities across different treatment modalities, including SPINRAZA, gene therapy, and new antisense oligonucleotides (ASOs) through research and collaborations:

  • Devices for administration: We are examining devices as a way to improve and ease intrathecal delivery of SPINRAZA, particularly for patients with complex spines. We have ongoing early research and are exploring possible research collaborations.
  • Gene therapy: Through our collaboration with the University of Pennsylvania, we have made substantial progress in a short time with this potential new treatment, with a goal of treating the first patient mid-2018.
  • Collaboration with Ionis: Initiated in December 2017 to explore new ASOs for SMA, this program is making good progress in identifying future ASOs.

The opportunity to make SPINRAZA available to even more patients in the US and around the world, as well as discovering potential new treatments, would not be possible without the support of the SMA community.

We deeply appreciate your contributions – and participation in ongoing clinical trial programs – that help shape the scientific community’s research in to and understanding of SMA. We look forward to sharing updates on our emerging and existing programs in future communications.

Best regards,

The Biogen SMA Team