FDA grants accelerated approval of tofersen for treatment of SOD1-ALS

The Food and Drug Administration granted accelerated approval of the drug tofersen for the treatment of SOD1-ALS. Developed by Biogen and Ionis, tofersen is an antisense oligonucleotide (ASO) that lowers SOD1 levels in the cell and, hopefully, can help to slow ALS progression.

The approval requires the companies to further study tofersen and verify its clinical benefits; if the additional trial confirms those benefits, the FDA can grant traditional approval for the drug. “The findings are reasonably likely to predict a clinical benefit in patients,” the agency said in a press release.

The development of tofersen stemmed, in part, from the early fundamental research by Packard investigators and members of the Center’s scientific advisory board Don Cleveland, PhD and Timothy Miller, MD, PhD who made the critical discoveries and model systems necessary to develop toferson as the candidate therapy.

“I’m delighted with this FDA approval and the reality that we have a truly effective genetic therapy for ALS patients with SOD1 mutations,” noted Jeffrey Rothstein, MD, PhD, Packard Center founder and director. “This approval has set the stage for the future of neurology treatment- eventually treating patients with this terrible mutation before actual disease manifests- with the great hope that the disease may never become evident.”

This week in Lille, France, Cleveland and Miller will receive the annual Rainwater Prize for Outstanding Innovation in Neurodegenerative Disease Research. The Rainwater Prize Program recognizes scientific progress toward new treatments for neurodegenerative diseases.

Additional Information about tofersen:

Read the FDA’s press release here: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene

To register for NEALS tofersen update webinar: https://register.gotowebinar.com/register/4854826952762535514

To register for the ALS Association tofersen patient update webinar: https://register.gotowebinar.com/register/4854826952762535514

Frequently asked questions about tofersen: https://www.als.org/navigating-als/living-with-als/fda-approved-drugs/tofersen

From the Packard Center archives: https://www.packardcenter.org/news-and-media/newsletters/preliminary-clinical-trial-results-for-sod1-als-look-promising