New Drug Treatment Shows Significant Benefit for People with ALS

These days, it seems everything is “news.” Or at least there needs to be enough “news” to fill the 24 hour news channels, your Facebook and Twitter feeds, and who knows how many website devoted to covering the “news.”

But in a time when everything claims to be “news,” it can be hard to tell when something genuinely newsworthy occurs. When we really ought to break out the “Breaking News” banners and pay attention.

For the ALS community, just such an event took place earlier this month, when the New England Journal of Medicine published encouraging clinical trial results showing that Amylyx Pharmaceuticals’ AMX0035 brought statistically significant benefit to people living with ALS. The study showed that AMX0035 decreased the rate of decline in the Revised ALS Functional Rating Scale (ALSFRS-R). The trial included 137 people with ALS and was conducted across 25 top medical centers through the Northeast ALS (NEALS) consortium.

People with ALS who received AMX0035 performed 2 points better on the ALSFRS-R compared to those who received the placebo. This is a statistically significant result, and in the real world could mean the difference between a person with ALS being able to feed themselves versus being fed, or the difference between needing a wheelchair versus not needing one.

Equally encouraging is that AMX0035 is an oral therapy dissolved in water that combines two existing drugs, sodium phenylbutyrate and taurursodiol. The safety of these two underlying drugs has been well established clinically, and the drug combination has been shown to be safe and well-tolerated.

And while this is great news now, what it could mean for the future is even more exciting for people with ALS and their families. In hopes of bringing this promising treatment to as many people as soon as possible, The ALS Association and I AM ALS have called on the drug company and the FDA to make the treatment widely available as soon as possible. Specifically, they ask that Amylyx submit a New Drug Application (NDA) for AMX0035 to the FDA for approval, and that the FDA review and approve that NDA swiftly. Further, they ask that the FDA require post-approval studies of AMX0035 on safety and effectiveness in the real world. In short, the hope is that AMX0035 can be made available to as many people with ALS as possible while phase 3 trials are underway, not after they are completed.

You can add your voice to those calling on the FDA and Amylyx to work together to bring AMX0035 to people with ALS as soon as possible. If you have not already, join the nearly 40,000 who have signed this petition asking for action now.

“The data clearly show that this drug should be a treatment option for everyone with ALS. We look forward to working with Amylyx, the FDA and the ALS community to make that happen as soon as possible,” ALS Association President and CEO Calaneet Balas said. “The FDA has the statutory authority to be flexible when reviewing drugs that meet criteria for safety and show promise for people with terminal diseases. This authority, combined with the ALS community’s expressed willingness to accept risk for potential treatment benefit, should compel the FDA to work quickly and cooperatively with Amylyx to make AMX0035 available to the entire ALS community without delay.”

ALS does not stop, and neither do we. While AMX0035 has the potential to improve the lives of many people with ALS, ALS researchers continue to search for even more treatments, and a cure. Your support has made this promising news possible. The ALS Association was able to financially support AMX0035 research through grants made possible by the Ice Bucket Challenge. Thank you for all you do, and for reminding us that we can’t wait in our efforts each and every day.

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