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Below is an update on Biogen’s tofersen clinical program.
About Biogen’s Investigational Therapy, Tofersen, for SOD1-ALS
Tofersen is an investigational molecule for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). A mutation in the SOD1 gene is believed to be a genetic driver of disease in approximately two percent of all ALS patients.
The safety and efficacy of tofersen is still being investigated in a Phase 3 placebo-controlled clinical study. This ongoing study includes patient volunteers living with SOD1-ALS, and at this time all participants enrolled are fast progressors. Successfully completing this study is the fastest and most certain way to file for global regulatory approvals and ensure access for SOD1-ALS patients around the world. In this study, some participants receive tofersen and other participants are randomized to placebo.
We designed our Phase 3 study to provide the scientific answers necessary to file applications for regulatory approval as quickly as possible and to expose as few patients as possible to placebo. For these reasons, the study is short in duration and includes only a small number of patients. Every single study participant in our study is critical to confirming the safety and efficacy of tofersen. Establishing a favorable safety and efficacy profile for a new medication is paramount and is essential to obtaining approval.
Access to Tofersen
Since our Phase 3 tofersen study completed its enrollment at the end of 2020, we have heard and listened to the voices of many patients with SOD1-ALS and their families seeking to gain access to tofersen before the study is completed and before tofersen is confirmed to be safe and effective.
Answering questions about access for new medicines outside of clinical studies is neither simple nor fast. Among the ethical imperatives in any access program are assuring that all patients receive equal treatment and priority and preserving the integrity of ongoing studies. Addressing these imperatives takes time and requires careful thought.
To respond to all these questions equitably and ethically, Biogen has engaged with regulators, clinical investigators, ethicists, and patient advocates to explore all potential ethical avenues to provide access to tofersen, outside of the ongoing Phase 3 study.
We do not believe it is fair to ask participants in this study to continue to receive placebo while other SOD1-ALS patients are offered access to tofersen, but we do believe that access could be provided as soon as the placebo-controlled study has ended. However, until the safety and efficacy have been established, we will prioritize early access for a subset of the most rapidly progressing patients with this rare, severe disease.
A Two-Part Tofersen Access Program
Beginning in mid-July 2021, after patients in this study currently on placebo transition to active therapy, and before the safety and efficacy of tofersen are established, compassionate use access will be allowed for a subset of the SOD1-ALS population with the most rapidly progressive disease. In the fall of 2021, if results from the Phase 3 study indicate that tofersen is safe and effective, and if no further studies are required, we will initiate an early access program (EAP) for the broad SOD1-ALS population.
Part 1: Individual Compassionate Use Access for the most rapidly progressing patients
Targeted start date: Mid-July 2021
As soon as participants in the Phase 3 clinical study are no longer randomized to placebo and have transitioned to tofersen, we will offer Individual Compassionate Use Access for the most rapidly progressing patients.
Access will start before we have completed analysis of the study data and therefore, before the drug has been confirmed to be safe and effective.
Eligibility criteria were developed in consultation with independent experts and bioethicists; access will be provided to SOD1-ALS patients with an ALSFRS-R slope decline greater than or equal to 2 points per month.
Confirmation of eligibility will be performed in an anonymized manner by a third-party organization.
Participation in this individual compassionate use access program will be available only in countries where it is permitted by local regulations and where we can ensure future access.
Individual access requests must be submitted by a patient’s treating physician.
Part 2: Early Access Program for the broad SOD1-ALS population
Targeted start date: Fall 2021
If the Phase 3 study data are positive and no further controlled clinical studies are required, we will immediately open an Early Access Program for the broad SOD1-ALS population in countries where this is permitted by local regulations and we can ensure future access.
This program will start before submitting applications for regulatory approval.
Biogen’s Commitment to the ALS Community
As you know well, Biogen’s quest to defeat ALS spans decades. While we hope this study’s results will be positive, we know firsthand that promising drugs can fail in Phase 3 studies.
We are committed more than ever to advance ALS research and to work alongside the community to evolve our processes to meet the needs of people impacted by ALS.
Learn more about Access Programs
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