Early Access

Update as of April 25, 2023

Tofersen is an antisense asset being evaluated for the potential treatment of SOD1-ALS. In the Phase 3 VALOR study, the primary end point as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) did not reach statistical significance; however, signs of reduced disease progression across multiple secondary and exploratory end points were observed. Based on totality of the data from the Phase 3 VALOR study and its open-label extension, Biogen is actively engaging with regulators, the medical community, patient advocacy groups, and other key stakeholders around the world to determine potential next steps.

In light of the critical unmet medical need, Biogen has provided access to tofersen through an early access program (EAP) since July 2021, in countries where such programs are permitted by local regulations and future access may be secured. EAP programs enable participants to gain access to a medicine free of charge before the treatment is licensed commercially. If a clear path forward for tofersen is not established, or if another controlled trial is required by regulators, Biogen may revise or discontinue the EAP. In April 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval for tofersen. Following this approval, the EAP closed to new US participants. In countries outside of the US where access has been available, the EAP remains open to new and ongoing participants.

Treating HCPs can submit requests on behalf of people with SOD1-ALS who meet the inclusion/exclusion criteria by emailing MedicineAccess@clinigengroup.com.

Update as of April 22, 2022:

Aducanumab is an Alzheimer’s disease treatment approved in the United States (U.S.) under the accelerated pathway based on reduction in amyloid beta plaques observed in patients treated with aducanumab. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). Aducanumab is still under review in countries outside of the U.S. Making aducanumab broadly available to qualified patients through regulatory approval is one of our top priorities.

Biogen recognizes the high unmet medical need of Alzheimer’s disease patients in countries where aducanumab is not yet approved. As we work with regulatory authorities, we are committed to offering access programs for eligible patients, where permitted by local regulations. There are two potential early access pathways to aducanumab for appropriate patients in some countries outside of the U.S. (early access to aducanumab is not available in the U. S. as the product is commercially available): an EAP and a Charged Managed Access Program (CMAP). We are working to assess the feasibility of the implementation of these programs on a country-by-country basis.

Effective April 22, 2022, Biogen has withdrawn its Marketing Authorization Application (MAA) for aducanumab from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) following interactions with CHMP indicating that the data provided thus far would not be sufficient to support a positive opinion on the marketing authorization of aducanumab by the EMA.

As a result, the aducanumab EAP will be closed in countries in the European Union that fall under the EMA jurisdiction, and no new requests for access to aducanumab will be accepted in those countries.

Biogen will continue to advocate for patients, and remains committed to research and development as well as to its collaboration with Eisai, working toward the goal of bringing new treatment options to people living with Alzheimer’s disease.

All access programs are subject to local regulatory and health authority requirements, and a patient’s physician is responsible for deciding if treatment with aducanumab is appropriate.

For information regarding the availability, access, and timing of these programs, a patient’s treating physician can send an email to medicineaccess@clinigengroup.com.

A qualified, licensed treating HCP may submit a request for early access to our investigational medical products, discuss eligibility, or ask other questions by submitting an online form. Please contact us at medical.communications@reatapharma.com for information prior to submitting a request for early access to therapy outside of a clinical trial. We will use our best efforts to acknowledge each submitted request within 2 business days after receipt of the written request and the completed application documentation. The request will be promptly evaluated after such acknowledgement in compliance with local laws and regulations.