Guide to Submitting Written Testimony to the FDA about NurOwn

On September 27, 2023, the FDA will host a public advisory committee meeting (AdComm) for BrainStorm Cell Therapeutics’s stem cell therapy NurOwn for ALS. At the AdComm, a panel of experts will review and evaluate the data provided by BrainStorm about NurOwn’s safety and efficacy and provide input that will help inform the FDA’s ultimate decision about NurOwn’s Biologics License Application. 

The deadline for FDA’s approval decision (the “PDUFA” date) on NurOwn after this AdComm is currently set for December 8, 2023.

The FDA invites people to submit written comments about NurOwn to the AdComm panel for their review prior to the September 27th meeting. People must submit their comments by September 20th for them to be included in the packet of materials provided to the panel.

In your written statements, please refer to “NurOwn” as “debamestrocel,” its technical name.

How to submit:

1. Go to https://www.regulations.gov/document/FDA-2023-N-2608-0001
2. Click on “Comment.” This blue button is the first one underneath the title. Please see the screenshot directly below.
   
3. In your comment, you must include:
   Docket No. FDA–2023–N–2608 for Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments
4. Write your comment. We have provided key points below to help.
5. Under “What is your comment about?,” choose “Individual Consumer.”
6. Attach photos or videos to your comment. This is optional. If you do this please remember that you can attach up to 20 files, but each file cannot exceed 10MB.
7. Enter your email address in the proper space.
8. Under “Tell us about yourself! I am…*,” click “individual.”
9. Click on the ReCaptcha box to prove you’re not a robot.
10. Click “Submit”
11. You did it!

The deadline to submit written comments that will be included in the official meeting packet of materials shared with the Advisory Committee members is September 20, 2023.

Comments submitted after the deadline of September 20th may be considered by the FDA but will NOT be included in the formal packet of materials provided to the Advisory Committee members.

Key points:

This is your time to make the case for NurOwn’s approval. Your goal here should not be to convince the AdComm that NurOwn works. BrainStorm’s presenters will cover the science. Our collective focus is on turning that data into stories about the real people we know are living and dying with the need for treatments and cures. We aim to humanize the science, bring home the message about what ALS really does as a disease, and why slowing, halting, or reversing symptom progression is critical. The AdComm panel may include individuals who are not experts in ALS or neurology and likely have not experienced ALS in their own family. We need to paint that picture.

Here are some key considerations to help make your comments impactful:

• Recognize the FDA’s consistent regulatory flexibility in all ALS drug approvals and your firm hope that policy continues when they evaluate NurOwn.
• Ask them to consider the totality of the evidence, the human experience, the state of ALS treatments, the limitations of the ALSFRS-R scoring system, and the science, including biomarkers.
• AdComm committee members might not understand the ALSFRS-R scale. Explain to them what a one point increase on the scale means –  physically, emotionally, and socially.
• Share your personal experience with ALS and how it has impacted your life or that of your family, friend, or loved one. 
• Explain why preserving a person living with ALS’ functionality (ability to do things independently) is important. 
• If you wish to incorporate science into your comment, please see: https://brainstorm-cell.com/publications_articles/ and https://brainstorm-cell.com/pipeline/ 

Other key points for your comments:

• ALS is a 100% fatal disease that often rapidly progresses.
• There are no therapies that halt or reverse symptom progression. Explain why a reversal of symptom progression, even in a few people living with ALS, is a huge achievement.
• This lack of therapies is the reason why any progress is a win – we are looking for progressive improvements while we work towards cures.
• There is a critical unmet need for therapies that slow down progression and give people more time and functionality.
• The ultimate goal for everyone with ALS is to live and have more time. We suggest sharing the personal hope you have with each new ALS treatment including the possibility of Nurown and the impact it will have for your or your loved one.
• Many people have died waiting for a decision to be made regarding NurOwn as a treatment for ALS, so a fair and transparent discussion of all the data is extraordinarily important to the ALS community.

Key phrases:

AdComm committee members will be presented with a lot of information. Repeating key phrases across comments might help AdComm committee members remember important facts as they make their recommendation. Here are some phrases that we suggest you use:

• “ALS is a 100% fatal disease.”
• “Any increase in someone’s ALSFRS-R score is an incredible achievement.”
• “Please consider the totality of evidence.”
• “Please continue the FDA’s pattern of regulatory flexibility of ALS therapies, so more people can live longer with ALS as we work towards a cure.”

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

https://www.regulations.gov/commenton/FDA-2023-N-2608-0001