RARE Daily

Biogen Will Provide Early Access to Experimental ALS Drug

April 27, 2021

Rare Daily Staff

Biogen, in a letter to the ALS community, said it will provide early access to its experimental ALS drug tofersen for patients with a specific mutation underlying their condition, but only after it has completed its current late-stage trial.

The decision is a reversal for the company, which had previously denied early access to patients who requested it. It said it will wait for the completion of the phase 3 study to do so out of fairness to trial participants assigned a placebo.

“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,” the letter said. “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.”

Tofersen is an experimental drug for patients with 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 2 percent of all ALS patients.

The safety and efficacy of tofersen is still being studied in a phase 3 placebo-controlled clinical study. This ongoing study includes patient volunteers living with SOD1-ALS. The company said all participants enrolled at this time are fast progressors. In this study, some participants receive tofersen and other participants are randomized to placebo.

“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,” the letter said. “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.”

The company said in the fall of 2021, if results from the phase 3 study indicate that tofersen is safe and effective and no additional studies are required, it will initiate an early access program for the broad SOD1-ALS population. It will also open an early access program for the broad SOD1-ALS population in countries where permitted and it can ensure future access.

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