Guide to Submitting Written Testimony to the FDA about Tofersen

On March 22, 2023, the FDA will host a public advisory committee meeting (AdCom) about Tofersen. At the AdCom, a panel will review and evaluate data provided by Biogen about Tofersen’s safety and efficacy and provide input that will help inform the FDA’s ultimate decision about Tofersen’s New Drug Application. The deadline for the approval decision (the “PDUFA” date) is now set for April 25, 2023.

The FDA invites people to submit written comments about Tofersen to the AdCom panel for their review prior to the March 22nd meeting. People must submit their comments by 11:59pm ET on March 8th for them to be provided to the panel for consideration.

This is your time to make the case for Tofersen’s approval. Here are some tips to help make your comments impactful:

• Explain how ALS affects you and your loved ones.
• If possible help explain the urgency for action and the “ALS CLOCK” how time is so urgent for people with ALS and their families to help improve functionality and extend life and what those days, weeks and months may mean to you and your family.
• Share how urgent is is to bring new treatments to market for people living with ALS & the tremendous potential this has for moving all science forward. “A rising tide lifts all boats.”
• Explain how critical this new product is because if will be the first drug that targets a genetic cause of ALS – superoxide dismutase 1 (SOD1 ALS).
• This drug will be the first that can slow or even stop the most aggressive version of ALS and will create new opportunities for more treatments for the entire community and help us finally make ALS a chronic disease rather than a 100% fatal disease.
• New data shows a clinically meaningful benefit in individuals living with SOD1-ALS.
• Data demonstrates that an earlier start of Tofersen slowed decline for individuals across efficacy endpoints (ALSFRS-R, slow vital capacity (SVC)).
• Data suggests a lower risk of death or PV when Tofersen is initiated earlier in treatment for an individual.
• The unmet medical need for people living with ALS is critical. This community needs therapies that work. The data shows that Tofersen is safe, effective, and we have hope that the FDA acts with urgency to approve this product for ALS.

Your perspective on the data and expertise in how ALS impacts people are invaluable. Please consider submitting written comments here.