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Biogen to release Tofersen BIIB067 July 2021 for “rapidly progressing”
Everyone,
In a press release yesterday, April 26, 2021, Biogen announced that it would make available in July 2021 its anti-SOD1 therapy Tofersen. The product release comes under Pres Trump’s Right To Try act. Details are below.This therapy is applicable to only 2% of all ALS cases. SOD1 occurs in only 15% of familial (inherited) ALS cases, and familial cases represent about 10% of all ALS cases (15% x 10% = 1.5%, less than 2%).
For details on how Tofersen works, and the history of its revisions and performance go to https://endals.altervista.org/#biib067 .
The drug is not a cure, but it may slow disease progression.When a cure is developed, it may happen first in SOD1 disease because the cause is known – and such large research companies, like Biogen, are involved. In other ALS variations, the primary cause is not known, not even the path to ALS development (or even whether this is just one path or many paths).
YusufBiogen to Make Experimental ALS Drug Available to Dying Patients
Tofersen was previously known as IONIS SOD1Rx, ISIS 333611 and BIIB067By Michelle Fay Cortez and Angelica LaVito April 26, 2021, 5:13 PM EDT https://www.bloomberg.com/news/articles/2021-04-26/biogen-to-make-experimental-als-drug-available-to-dying-patients
• Compassionate-use program for Tofersen will start in July
• Early-access move comes after patients pushed for treatmentBiogen Inc. said it will make an experimental medicine for Lou Gehrig’s disease available to some people who are dying of 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 healthcare 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.
Welcomed Move
Biogen’s decision was welcomed by advocacy groups who said the company listened to the patients it was trying to help.
This is the power of those impacted by ALS like @Chance4Lisa to have their voices heard. Thank you @biogen for listening to the ALS community and taking action to get people living with ALS access to this therapy sooner than otherwise planned. https://t.co/QaTYa26Yf2 https://t.co/bt1VlLn37T
— I AM ALS (@iamalsorg) April 26, 2021</p>
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 are 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.”
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1 Reply
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This is one of the treatments coming out of the Pre-fALS study. I attended a zoom seminar on the next study of Tofferson. It is looking promising for the pALS with one of the SOD1 mutations. I’m very excited to see what happens with this study! It is awesome that they are considering making it available to people outside the study.
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