Fact checked byHeather Biele

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December 05, 2022
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European Medicines Agency accepts marketing authorization of ALS treatment

Fact checked byHeather Biele
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Biogen announced that the European Medicines Agency has accepted the marketing authorization application for tofersen, an investigational drug for the treatment of superoxide dismutase 1 ALS.

According to a company press release, superoxide dismutase 1 (SOD1) ALS is a rare, progressive and fatal disease with no treatment, affecting approximately 2% of people with ALS. The antisense oligonucleotide drug tofersen has been designed to bind to SOD1 mRNA, prompting its degradation and reducing SOD1 protein production.

Source: Shutterstock.com.
Biogen has announced that the European Medicines Agency has accepted the marketing authorization application for tofersen, an investigational drug for treatment of superoxide dismutase 1 ALS. Source: Adobe Stock

The marketing authorization application includes results from the phase 3 VALOR study, the release stated, which in September were published in the New England Journal of Medicine.

Timothy Miller

“The SOD1 protein was reduced in the cerebrospinal fluid,” Timothy Miller, MD, PhD, the study’s principal researcher, told Healio. “This shows that tofersen is hitting its target; it is reducing SOD1 protein. This is not a surprise, but it is good news to show that it does indeed do what it is supposed to do.”

In addition to reducing SOD1 protein production, Miller reported that tofersen also lowered levels of neurofilament light protein.

“Neurofilament is an intermediate filament protein that is in neurons, and it leaks out of the neurons when they are damaged,” he said. “It is not specific — it leaks out in many neurodegenerative diseases, including ALS. But the fact that it was substantially lowered in this study, my interpretation of those data are that we have substantially slowed down the neurodegenerative disease process.”

According to the release, tofersen is also under review by the FDA after the agency accepted a new drug application for the treatment in July. A decision from the FDA is due in April 2023.

Philip Van Damme

“For such a long time, we have not had any submissions do enough for effective, or potentially effective, drugs for ALS,” study researcher Philip Van Damme, MD, PhD, professor of neurology and director of the Neuromuscular Reference Center at the University Hospital Leuven in Belgium, told Healio. “This is an important milestone — and now the formal evaluation process can start.”