May 4, 2017
Dear members of the SMA community,
In response to your request for information, we would like to provide you with an update on Biogen’s global Expanded Access Program (EAP), called the Special Access Program (SAP) through Health Canada.
Based on the high unmet need in SMA, and to bridge the gap between the closure of the sham-controlled phase 3 ENDEAR study and regulatory approval across Europe and Canada, Biogen is sponsoring an EAP for eligible individuals with infantile-onset SMA (most likely to develop Type 1). In Canada this is via the Special Access Program through Health Canada. This program enables wide access to nusinersen, subject to applicable inclusion criteria and as permitted according to local laws and regulations, during the time before a potential regulatory approval.
Opening an EAP in infantile-onset SMA has taken longer than expected in many countries due to the operational complexities in opening and running a program like this in a previously untreated disease. Despite this, as the demand from individuals with infantile-onset SMA increased, and in response to requests from clinicians, we are currently further expanding the nusinersen infantile-onset EAP to multiple countries. This expansion is subject to local laws and regulations, where it can be operationalized and where there is a path to long-term availability and reimbursement of nusinersen. Even with this expansion, there are still many more infants to be treated.
Given these complexities and the anticipated regulatory approval in the EU and Canada, Biogen’s global EAP will not be extended beyond the infantile-onset (most likely to develop Type 1) SMA population. We will continue to focus on the urgent treatment of individuals with infantile-onset SMA, based on the severity and high risk of mortality and the ability for centers to meet the current care requirements for these patients. We understand this decision will not meet the needs of the entire SMA community; however obtaining regulatory approval across the globe remains our number one priority, as we believe it is the best route to long-term sustainable access to nusinersen for the greater SMA community.
At the time of Health Canada approval, the steps to achieve reimbursement will progress according to Canadianspecific requirements. Biogen Canada will continue to work with agencies and provincial governments through the required processes of review and approval so that Canadians with SMA can have access to nusinersen.
Patients currently enrolled in the SAP program in Canada will remain in the program until Health Canada approval. Following Health Canada approval the SAP program will close however, Biogen Canada will continue to provide nusinersen to patients already receiving nusinersen via the SAP at the time of Health Canada approval. Once Health Canada approval has been secured, Biogen will continue to work towards securing reimbursement for all SMA patients. Obtaining regulatory approval across the globe remains our number one priority, as we believe it is the best route to long-term sustainable access to nusinersen for the greater SMA community. We continue to work with Health Canada to conclude our discussions.
We are thankful to the entire SMA community for your continued support, and in particular, the individuals and families who have participated in our studies and supported the nusinersen program. We remain committed to transparent and timely communications and will continue to be available to provide requested updates.
For further questions, please contact Biogen Canada Inc Medical Information at: 1-866-477-3462