A pedestrian walks past Biogen Inc. headquarters in Cambridge, Massachusetts, U.S., on Monday, June 7, 2021.
Adam Glanzman | Bloomberg | Getty Images
The Food and Drug Administration’s independent panel of advisors on Wednesday voted against the effectiveness of
“The trial that was presented unfortunately did not meet the primary and secondary endpoint,” said Dr. Liana Apostolova, a professor of neurology at Indiana University School of Medicine who voted against tofersen’s effectiveness.
But the panel voted unanimously that the drug could have a clinical benefit in reducing a protein that is associated with disease severity.
Michelle Mielke, a professor of epidemiology at Wake Forest University School of Medicine who voted in favor of the drug, acknowledged the data isn’t fully conclusive but said “there are several aspects of the data that do suggest strong clinical evidence.”
“And again, my decision also weighed in the fact that there really is an unmet need,” she added.
Accelerated approval is an FDA designation that clears drugs faster if they fill an unmet medical need for serious conditions. Such an approval would require Biogen to study the drug further to verify its clinical benefits.
The FDA typically follows the advice of its advisory committees but is not required to do so. It will make a final decision on April 25.
ALS, most commonly known as Lou Gehrig’s disease, is a progressive and fatal neuromuscular disease that causes nerve cells in the brain and spinal cord to waste away over time, causing people to lose control of muscles needed to move, speak, breathe and eat. The disease eventually causes paralysis and even death, and generally affects people between 40 and 70 years old.
The drug targets a form of ALS in people with mutations in a specific gene passed down through generations within families. Those mutations can cause a protein called SOD1 to accumulate to toxic levels, which can ultimately damage the nervous system and lead to the development of ALS.
Only a few thousand people worldwide have been diagnosed with that kind of SOD1 mutation, or around 2% of the 168,000 people who have ALS globally, according to Biogen. That number is even smaller in the U.S., with roughly 330 people affected by the SOD1 mutation. The median survival time from diagnosis with the rare form of ALS to death is 2.7 years, according to the company.
The SOD1 mutation is associated with 20% of cases that occur within families.
Families impacted by ALS hope the drug could pave the way for more research on how to target the cause of disease, potentially leading to new treatments for the estimated 5,000 new people in the U.S. who get diagnosed with ALS every year. Globally, researchers from the National Institutes of Health expect ALS cases to increase by nearly 70% to around 376,000.