A Win for AMX0035 - What does this mean for you?

Danielle Geraldi-Samara, MD

September 30, 2022

Danielle Geraldi-Samara, MD
February 9, 2024

On September 7, 2022, we heard a collective “YES!!” from the ALS community as the FDA advisory committee voted 7 – 2 in support of the approval of the medication known as AMX0035 for the treatment of ALS.  AMX0035 is an oral combination medication of sodium phenylbutyrate and taurursodiol which protects the inner workings of the neurons and may slow the progression of ALS. So what happens now and what does this mean for you?

Step 1: Approval. This advisory committee’s recommendation is not the same as the FDA granting approval. For that to happen, the FDA will review the recommendation and data, and will deliver its final decision, which we anticipate will come by September 29, 2022.

Step 2: CMS. If the FDA approves the medication, physicians will then be able to prescribe it–but this does not mean it will be immediately commercially available. That availability will depend upon the pricing of the medication, as well as review of that pricing by Centers for Medicare & Medicaid Services (CMS). Pricing of AMX0035 is currently unknown. As for CMS, the review can be as fast as a month or can take a few months, depending upon the pricing and the complexity of the review.

Step 3: Other Insurers. As you know, CMS is in charge of overseeing Medicare and Medicaid. After they conduct their review, each private payer (e.g., Aetna, United, Blue Cross Blue Shield, etc.) will conduct its own pricing review to determine what price it will allow and whom it will cover for the medicine. Right now, the data suggests that the medication may benefit all adults with definite sporadic or familial ALS. That being said, there may be some additional qualifiers based on certain respiratory measures and time to symptom onset.

Step 4: Production. While the pricing review is underway, the company Amylyx will have to increase their ability to produce AMX0035 at a commercial level. The amount of time that takes will vary depending upon the type of drug, as well as how hard it is to produce. Here, we hope that Amylyx will be able to ramp production quickly, as the drug is an oral medication and not an intrathecal injection.

Step 5: Coverage. I wish it was different, but what a physician deems as an eligible patient does not mean that all insurance companies will agree.  Some insurance companies may put this medication on their formulary while others may not.  This is not something we can predict, but certainly something that Synapticure can help you with, from working with our care coordinators to determine optimal insurance providers to obtaining prior authorizations, applying for grants, or appealing denials of coverage. Synapticure can also work directly with the company to expedite obtaining the medication.

These are typical bumps in the road for any new medication that comes to the market, but we understand the great importance this holds for YOU, as this is only the third medication that is FDA approved for ALS.  In light of that, we are committed to exhausting all avenues to obtain the medication as soon as possible, if you and your physician decide AMX0035 is the best treatment course to take.

Photo credit: Fayette A. Reynolds, Berkshire Community College Bioscience Image Library

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