Biogen has provided the following community statement on the final results from CHERISH, a Phase 3 study of SPINRAZA.
Dear Members of the SMA community,
This past December marked one year since the FDA approval of SPINRAZA® (nusinersen), the first and only treatment option for children and adults living with SMA in the U.S. Since then, SPINRAZA has received regulatory approvals across the globe, including the EU, Japan, Brazil, Canada, Australia, Switzerland and South Korea. We’re proud to share that more than 3,200 individuals with SMA are being treated with SPINRAZA worldwide.*
We are also pleased to share that the final results from CHERISH, a Phase 3 study of SPINRAZA, were published in The New England Journal of Medicine (NEJM). The full manuscript, titled “Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy,” appears in the February 15, 2018 publication of NEJM. Publishing this information is part of our continued efforts to provide robust clinical trial data that support the favorable efficacy and safety profile of SPINRAZA and support for broad access to the therapy for those who may benefit.
Biogen is incredibly thankful to the entire SMA community for their continued support, contributing to both the many regulatory approvals within the last year and the ongoing clinical trial programs. The support of the community has helped thousands of individuals with SMA receive ongoing treatment around the world. Biogen is continuously assessing the possibility of expanding access of SPINRAZA across the globe. Although Biogen may not have plans to bring SPINRAZA to market in every geography we are exploring alternative options for families living with this devastating disease.
We continue to recognize that there is more work to be done to ensure children and adults living with SMA may receive access to SPINRAZA, and we are working on the following to help overcome the current challenges:
- Supporting the growing number of treatment delivery sites: There are now more than 215 sites that have started administering SPINRAZA in the U.S. Physicians and advocacy groups continue to work toward increasing capacity and adding additional sites to administer treatment for pediatric and adult patients.
- Addressing intrathecal access challenges in those with spinals rods and/or fusions: We are continuing to partner with specialists including interventional radiologists, spinal surgeons, neurologists in identifying approaches and techniques best suited to deliver SPINRAZA to all patients who may require treatment. We are also evaluating device collaborations but these are very early efforts and we are working to determine a potential path forward.
- Advancing plans for teens and adults: We are supporting initiatives and study proposals which can further demonstrate the value of SPINRAZA in this patient population. The CHERISH study is just one part of the largest well-controlled clinical development program conducted to date in the history of SMA, 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, which will provide a deeper understanding of SMA and SPINRAZA. As always, we will continue to partner with advocacy groups to ensure the community is aware of new trials as they become available.
Lastly, we continue our commitment to individuals with SMA and their families and our aim to increase the therapeutic options available to treat SMA. In December of 2017, we announced a collaboration with Ionis Pharmaceuticals, Inc. to identify new antisense oligonucleotide drug candidates. These efforts are in the early preclinical stages, but we will be excited to share more information in the coming years.
We thank everyone for their feedback, continued support and enthusiasm around SPINRAZA. We remain a dedicated partner to this community and will continue to provide updates in the future.
Thank you,
The Team at Biogen
*Based on commercial, Early Access Program and clinical trial participant patients as of December 31, 2017.