Access to investigational therapies
How to request access
- Types of access
- How to request access
For Patients / Caregivers / Patient Advocates
If you are a patient and would like to request to participate in an expanded access program (EAPs) and single patient access or emergency use, the licensed physician overseeing your care must submit this request.
For more information on this process for any of Biogen's investigational drugs, please contact the Biogen Patient Center. Requests submitted to this email address will be acknowledged within one business day.
For Healthcare Professionals
Listed below are Biogen’s current EAPs (note an EAP typically ends once the investigational therapy has been approved and is commercially available in the patient’s country):
- SPINRAZA™ (nusinersen)/ Spinal muscular atrophy (SMA)
Patients who are not eligible to access the above therapies in a commercial or clinical trial setting may be able to access them through an EAP. If you are a healthcare professional and would like to learn more, please contact our partner Idis Pharma.
For information about access to other Biogen therapies that are not available to patients in the commercial or clinical trial setting, please contact:
If you are a distributor or wholesaler and would like further information, please contact the Biogen Patient Center.
Biogen has opened a nusinersen expanded access program (EAP) for eligible patients with infantile-onset (consistent with Type 1) spinal muscular atrophy (SMA).
The program is now active; however, not all sites around the globe will be up and running at the same time. Sites can participate in the EAP in countries where EAPs are permitted according to local laws and regulations, can be operationalized and there is a path that can support long-term availability of nusinersen. Many sites are still working to operationalize the program at their institution. There will be variation both within and across regions as to when individual sites begin scheduling and dosing patients.
A doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
Per U.S. regulations, the EAP for nusinersen will close to new individual enrollment in the U.S. now that the FDA has approved the New Drug Application (NDA) for nusinersen.
Individual clinicians and the team at Biogen will assist individuals currently participating in the EAP in the U.S. to transition to the approved therapy. For more information, families currently enrolled in the EAP should an SMA Support Coordinator at 1-844-4SPINRAZA (1-844-477-4672).
In Europe, now that the European Commission (EC) has granted a marketing authorization for nusinersen, individuals currently enrolled in the EAP will continue to receive the medicine free of charge until local access agreements are completed. The EAP in Europe will remain open for new patients for a period of time following marketing authorization if allowed by local legislation. The actual date of EAP closure will be defined by Biogen on a country by country basis.
The EAP will continue in geographies where it is currently open and nusinersen is not yet approved by regulatory authorities.
Please review Biogen’s corporate EAP principles and criteria. Detailed information on the nusinersen EAP for eligible infantile-onset (consistent with Type 1) SMA, including eligibility criteria and participating sites, will be posted on clinicaltrials.gov in the coming weeks.
Patients who are interested in more information on this process should contact the Biogen Patient Center.
Physicians interested in more information should contact Biogen Medical Information.
(Posted June 1, 2017)