Dear members of the SMA community,
In response to your request for ongoing information, we are providing you with additional updates.
The main points in this update include:
• the progress being made in Europe and Canada to obtain regulatory approval for nusinersen,
• clarification around recent news about the U.S. price that was released late last year,
Our number one priority is to obtain regulatory approval as soon as we can. 2016 was an historic year for the SMA community. As announced in December, nusinersen received United States FDA regulatory approval for the treatment of all individuals with SMA in the United States.
Regulatory approval pathways in Europe and Canada follow a different process, with different timelines, compared to the U.S. As previously communicated, Health Canada is currently reviewing our file and we anticipate a response from the agency mid-2017.
We are providing all the data we have to date. Health Canada is reviewing the totality of the data available and will determine approval and the final nusinersen label which will identify which patients are eligible for treatment. If Health Canada approves nusinersen, we will be working with public (provincial) drug plans and private insurance companies in Canada to find a way forward to bring our medicine to the Canadian community. Biogen recognizes that cost and access to treatments are key considerations for patients, providers, payers, and policy makers and we are committed to working with health-care systems and governments to find solutions to bring nusinersen to patients in need.
The healthcare systems and pathways to access in Canada and the European countries are very different than the US. However, with the recent approval by the FDA and access within the US, we feel it is important to share the principles on which Biogen articulates its approach to prices of our medicines. Consistent with the pricing of the overall Biogen portfolio of therapies, we strive to achieve an appropriate balance among three key Biogen pricing principles – clinical value the therapy provides to patients and families, the impact this new treatment has on the healthcare system, and finally our ability to fulfill our long-term commitment to patients and advancing science through the funding of research and development. We will be using these principles to determine the European and Canadian price and be compliant with country-specific guidelines.
We want to assure you that we are committed to ensuring nusinersen is available to patients who may benefit from the medicine and have begun working, where feasible, with health-care systems and governments to discuss solutions for introduction as quickly as possible, should nusinersen be approved. We will continue to address your requests for more information.
Based on the unmet medical need in SMA and with a great sense of urgency, Biogen initiated a global expanded access programs (EAP) for eligible patients in infantile-onset SMA (most likely to develop Type 1) in September 2016, before filing with the regulatory agencies. This was initially initiated at existing nusinersen clinical trial sites in countries where EAPs are permitted according to local laws and
regulations, where it can be operationalized and where there is a path to long-term availability and reimbursement of nusinersen. Given the complexities and operational aspects of opening an EAP in SMA, the initial phase in infantileonset has taken longer than expected to open. Considering the high unmet medical need and sense of urgency to treat infantile-onset SMA the EAP program will continue to focus on these infants. In parallel, we will continue to evaluate the feasibility to broaden the EAP to include later-onset SMA (consistent with Type 2). Obtaining regulatory approval remains our number one priority, as we believe it is the best way to provide broad and sustainable access to the SMA community.
Finally, we had several questions in relation to clinical data and study results. For any specific, medical scientific questions related to nusinersen please refer to SMA experts in your area. We will be disclosing the details throughout the course of 2017 at medical scientific conferences and on public websites in accordance with local legal and regulatory requirements.
We appreciate the opportunity to share this information with the Canadian SMA community. We will continue to ensure that the perspectives of patients and families are reflected in every decision we make. And, of course, we remain committed to supporting the SMA community and partnering with healthcare systems to ensure that patients who may benefit from nusinersen will have access to therapy, once approved in Canada. We continue to understand the urgency for patients with SMA and their families.
For further questions, please contact Biogen Canada Inc Medical Information at: 1-866-477-3462