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In September 2022, the ALS community celebrated a major milestone: the FDA approval of a new treatment for the disease, Amylyx Pharmaceuticals’ Relyvrio. The drug, also known as AMX0035, and Albrioza in Canada, was shown to slow loss of physical function in people with ALS in a phase 2 trial completed earlier this year. A phase 3 trial in Europe is also currently recruiting. Relyvrio will now become the third treatment option for people with ALS in the US, following riluzole and edaravone.

While this is certainly a cause for celebration, it also raises many questions for people hoping to access the drug. How will the new drug affect ongoing and upcoming clinical trials? Will most insurance plans pay for it? And what is the best way to go about accessing the treatment?

To better understand the choices that people with ALS are facing, the ALS Therapy Development Institute (ALS TDI) spoke to several members of the ALS community about their concerns and expectations as the new drug comes on the market.


Gwen Petersen: What does Relyvrio approval mean for people with ALS?

Gwen Petersen, who is living with ALS, is an advocate who has served on many industry patient advisory boards in the pharmaceutical industry, as well as the I AM ALS Clinical Committee, NIH ALS Steering Committee, and Her ALS Story. Gwen shared her thoughts on why the Relyvrio approval is an exciting development for the ALS community, and what questions remain to be answered.

Generally speaking, as a person with ALS and an advocate, how are you feeling after the approval of Relyvrio (AMX0035)?

So, this approval is a win for a few reasons. One, I've been in the AMX0035 EAP for the last three months, and it's been going well. It's easy administration. It's essentially a drink every 12 hours. I haven't experienced any side effects. The one common side effect is diarrhea. No diarrhea here!

Then, the second reason I'm excited about approval is because, according to the study data, this is the most efficacious drug we have in ALS. It extends life longer than anything else FDA-approved at this moment. So, in terms of promise, this would have to be the most promising approved therapy for ALS right now.

If there's a third reason to be excited, I would love for researchers and doctors to start exploring how the approved portfolio of drugs – Amylyx, riluzole, and edaravone – interact with each other in combination. Do they extend life even more? There's a lot of potential now for this as the start of a cocktail.

Are you aware of any concerns or issues with insurers or prescribers that might interfere with access for Relyvrio for people with ALS currently?

We will see. It's a question I myself have. I think it'll be based on everyone's individual insurance. Like, what will Medicare do? Does Medicare have the infrastructure, do they have the bandwidth to approve all three ALS drugs and administer them? It's a top question of mine. I will be on all three. So, what's that going to look like? Cost is the question at the top of my list.

Do you have any other concerns about access to Relyvrio you’re hoping to see addressed soon?

Yes. Are our neurologists going to tell us that AMX is now approved and show us the path to access it? Are they going to lead us down the path, or will we need to do it ourselves? Do we need to initiate conversations with our care team to get on this? I would hope care teams will be proactive. Maybe that's an email from the clinic saying this has been approved. "If you're interested, please reach out to X, Y and Z." I would hope our care providers will be proactive, but I'm leery of that.


Dr. Nadia Sethi: What challenges might clinical trial participants face in accessing Relyvrio?

From the time that her husband felt his first symptoms of ALS, Dr. Nadia Sethi has been engaging with the ALS community, participating in, and leading groups to increase understanding of the science of ALS and the clinical research landscape. Dr. Sethi joined ALS TDI as our Director of Community Engagement and Outreach in 2021. She weighed in on the potential complications people in clinical trials might face in trying to access the drug.

Do you believe people in clinical trials that are running right now will be able to access Relyvrio?

Overall, I think it could be challenging for people in ongoing studies to access Relyvrio. I believe that sponsors will try to find the best path forward while preserving the scientific integrity of their trials. When trials are designed, sponsors must consider, within their statistical analyses, the effects of approved medications that participants might be taking. Introducing a new medication mid-study could make it harder to interpret data from the trial.

What about upcoming clinical trials that may already be designed, but are not recruiting yet?

For trials that are ongoing that people are now considering joining, they could face some important choices based on what matters most to them. They may have to choose between taking part in a clinical trial, where we do not understand the efficacy of a drug yet or taking a treatment that is newly approved. It is best for people considering trials to have open discussions with both their care team and trial site when weighing the risks vs benefits, as well as outlining their personal priorities. I know when going through ALS with my husband, we opted to take an approved treatment over taking part in a particular clinical trial that wouldn’t allow it, but that might not be the right decision for everyone.

What do you think trial sponsors can do to address these concerns?

Trial sponsors are aware of the difficult decisions people will face. Some are trying to accommodate the new approval where they can and allowing participants who move on to an open label extension (OLE) to take Relyvrio.

Also, a great example of a sponsor working to meet the needs of the ALS community is Sanofi, a company that recently started a global phase 2 trial for ALS. Sanofi just announced they will be amending their trial protocol to allow participants in the US and Canada to take Relyvrio.  


Kevin Robinson: What is the current reality of Relyvrio access for people with ALS?

Kevin Robinson is a retired Navy captain who served 41 years on active duty. In July 2021, he was diagnosed with bulbar onset ALS. He is currently a participant in the Healey Platform Trial enrolled in Regimen E: Trehalose. We spoke to Capt. Robinson about his experience working to gain access to Relyvrio – and how he made the decision to move forward with it.

In general, what has the process of trying to get access to Relyvrio been like so far for you?

The process has been somewhat painful. Bear in mind I have both VA Health (Optum) coverage, as well as TRICARE via Humana Military. Neither of these have been successful yet. Additionally, my ALS Clinic here in Virginia Beach is trying to work with insurance companies and Amylyx.

What is most frustrating are the inclusion/exclusion criteria insurance companies have either already adopted or are contemplating. Here is an example is the draft criteria from the VA. As you can see, there is excessively strict criteria such as:

“Any shortness of breath; Any difficulty breathing while lying flat; Any respiratory issues; Greater than 18 months since onset. Additionally, the inclusion criteria that one must be able to walk, and self-feed is excessively restrictive.”

As someone participating in the Healey Platform Trial, have you received any information about whether you'll be able to access Relyvrio?

I have received notice from Mass Gen Healey that I am able to take Relyvrio now that I am part of the OLE.

How have you been thinking about the decision of whether to pursue Relyvrio? Does the answer to question 2 affect your thinking at all?

I have struggled with the decision of whether I will pursue Relyvrio. Initially my concern was whether adding it to my regimen could affect the trehalose OLE data. Now that Healey has decided it is okay, that concern is allayed.

However, I also have more practical concerns about whether to pursue it. First, we are not talking about a home run type drug/therapy. While it has shown benefit in some, there were negatives associated with the trial as well. Second, I am concerned about possible ramifications of adding a new, relatively unproven drug to my regimen. I certainly understand and believe that ALS treatment will be a cocktail mixture of drugs/therapies. However, at what point are we just lobbing darts at a dart board?

Unlike many in this battle, I do not subscribe to the “more is better” philosophy. I have been on riluzole, Radicava, and Nuedexta for over a year. I just completed the placebo controlled portion of the trial and have now had my second OLE infusion. I have had little progression over the last year, with my ALSFRS-R being 42 or greater from 6 different neurologists. In discussing Relyvrio, each have said they would be hesitant to recommend any changes to my current course, given my relatively slow progression.

It is quite the conundrum: Do I add something that could reduce the effectiveness of my current regimen? Or do I stay the course with what may be working now? Of course, the difficult part of trying to answer those questions is the fact I am less than 2 years since symptom onset, which means I fall into the category of early patients where variability in progression can be significant.

ALS TDI would like to thank Gwen, Nadia, and Kevin for taking the time to speak with us and share their perspectives on Relyvrio. To learn more about Relyvrio, visit the Amylyx website at https://www.amylyx.com/product.


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