Biogen has provided the following community statement on Spinraza.
Dear Members of the SMA community,
As part of our commitment to the SMA community to provide ongoing and comprehensive communications about SPINRAZA® (nusinersen), we are pleased to share that the final results from ENDEAR, the Phase 3 study of SPINRAZA, were published in The New England Journal of Medicine (NEJM). The full manuscript, titled “Nusinersen Versus Sham Control in Infantile-Onset Spinal Muscular Atrophy,” appears in the November 2 issue of NEJM. Publishing this information is part of our efforts to provide additional details on clinical trial data that support the favorable efficacy and safety profile of SPINRAZA and support access to the therapy for those who may benefit.
The ENDEAR study is part of the largest well-controlled clinical development program conducted to date, which includes more than five years of data for SMA. Biogen and the physician community continue to collect and evaluate data through SHINE, the SPINRAZA open-label extension study, and we are planning to conduct new clinical research across different SMA populations. These efforts will provide a deeper understanding of SMA and SPINRAZA, and will support broad access over the long term.
We are incredibly thankful to the entire SMA community for their continued support, and especially the nearly 200 individuals and their families in the U.S. who have participated in our clinical studies. Their participation helped lead to the early and broad December 2016 FDA approval of SPINRAZA, allowing, as of September 2017, approximately an additional 1,200 U.S. patients to start on therapy, of which approximately two-thirds are individuals with Type 2 or 3 SMA.
However, we are keenly aware that there are many more who may benefit but have encountered challenges, including availability of local treatment sites, reimbursement delays, and for some patients, complexities arising from the intrathecal injection.
We continue to work with stakeholders so that people who can benefit from SPINRAZA may gain access to therapy. Our current and planned activities to help overcome these challenges include:
- Supporting the increasing number of local treatment delivery sites: There are now over 180 sites that have started dosing across the U.S. Physicians and advocacy groups are working to add more in the coming months, striving for a balance of both pediatric and adult sites.
- Educating stakeholders on outcome measures: Biogen believes that delivery of a high level of care in combination with the accurate and standardized use of measurement tools is an important part of managing SMA. We therefore continue to support SMA education initiatives for the community, including for physicians, physical therapists, and payers, with these goals in mind.
- Supporting physicians in addressing administration challenges: Biogen is actively engaging with the physician community, which has novel ideas regarding approaches to administering treatment for patients with complex cases, such as those with varying degrees of scoliosis and spinal rods/fusions. We are also working to understand how to make such innovations in spinal access available to more treatment centers in the U.S., where appropriate. While we realize there is an urgency to help find solutions, we also acknowledge this process may be lengthy, and Biogen is committed to finding the safest and most effective way to provide access to individuals with SMA.
Lastly, we recognize the ongoing efforts to add SMA to Newborn Screening (NBS) panels and we are proud to be a partner in this community-wide effort, with Cure SMA, other companies and you. We look forward to the upcoming federal decision by the Recommended Uniform Screening Panel (RUSP) in February 2018, and are pleased by the early efforts in some states, such as Missouri, Minnesota and Utah, leading to adoption of SMA in NBS panels.
One of the many things we’ve learned about the SMA community is that you are motivated and committed. Many of the challenges we originally identified when launching SPINRAZA are gradually being overcome – and that’s largely a reflection of the incredible determination of this community of providers, families and individuals with SMA.
We remain a dedicated partner to the SMA community and will continue to provide updates in the future.
The Team at Biogen
SPINRAZA 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 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 side effects of SPINRAZA include lower and upper respiratory infections, constipation, headache, back pain, and post-lumbar puncture syndrome.
These are not all of 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.
This information is not intended to replace discussions with your healthcare provider.
Please see full Prescribing Information for additional Important Safety Information.