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Biogen to make experimental ALS drug available to dying patients

Lauren Farley and her mother, Susan, march through Kendall Square on their way to Biogen during a rally on March 30 for their aunt and sister-in-law, Lisa Stockman Mauriello, who has been diagnosed with a rare and aggressive form of ALS. Her friends and supporters are beseeching Biogen to grant her compassionate use of a drug, tofersen, that could save her life.Jessica Rinaldi/Globe Staff

Biogen Inc. said it will make an experimental medicine for Lou Gehrig’s disease available to some people who are dying of the incurable illness starting in July, following months of pressure from patients who had no other treatment options.

The drug, known as tofersen, will be offered to the most rapidly progressing patients after researchers complete a key study this summer, the company said in a statement posted on its website.

Tofersen hasn’t been reviewed or approved by regulators in any country. It will be given on a compassionate-use basis after everyone who was given a placebo during the clinical trial has been offered the medicine, Biogen said.


The move comes after Lisa Stockman-Mauriello, a health care communications executive who was diagnosed with the disease also known as amyotrophic lateral sclerosis in January, launched an aggressive campaign to get access to the medicine under the Right To Try law that was signed by former President Donald Trump or through a conventional compassionate use program backed by the Food and Drug Administration.

Biogen refused to make the drug available to patients outside of ongoing studies while anyone who was participating in them was still taking a placebo, arguing that it would be unfair to them. No studies were available for Stockman-Mauriello or other patients to participate in at the time, giving them no avenue to access the medicine that was specifically designed for people with their exact genetic form of the disease.

Biogen’s decision was welcomed by advocacy groups who said the company listened to the patients it was trying to help.

The company plans to make tofersen available to those with the most rapidly progressing form of the deadly disease in July, after the final study is done and before the results have been fully analyzed.

If the medicine is found safe and effective in that trial, Biogen will offer a broader program for more patients even before it files for regulatory approval.


Taking the drug before its safety and effectiveness is established will be a risk for patients and for the company. It’s a gamble some patients have said they are willing to take. Now Biogen has concurred.

“While we hope this study’s results will be positive, we know firsthand that promising drugs can fail in Phase 3 studies,” the company said. “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.”