Amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease, is a neurological disorder that impacts the motor neurons responsible for controlling voluntary muscle movement and breathing, according to the National Institutes of Health (NIH). The Centers for Disease Control and Prevention (CDC) has reported that at least 16,000 Americans are affected by ALS. Among these cases, 90% are sporadic, occurring randomly, while the remaining 10% are inherited.
Today, we will be analyzing the latest guidelines on ALS and exploring any recent changes that could potentially influence the upcoming Clinical Practice Guidelines for ALS. There are two US-based guidelines for the management of ALS. Interestingly, both of these guidelines were initially published in 2009 and have since been reaffirmed by the American Academy of Neurology (AAN) in 2023:
- The Care of the Patient with Amyotrophic Lateral Sclerosis: Drug, Nutritional, And Respiratory Therapies
- The Care of the Patient with Amyotrophic Lateral Sclerosis: Multidisciplinary Care, Symptom Management, and Cognitive/Behavioral Impairment
Since the original publication date, there have been three new medications approved for patients with ALS. However, one of these medications, Relyvrio (AMX0035), produced by Amylyx Pharmaceuticals, was withdrawn from the market on April 4, 2024, due to unsatisfactory clinical trial outcomes. As a result, there are currently three approved medications for ALS:
FDA-Approved Drugs for ALS
| Approval Year | Drug | Company | Comment |
|---|---|---|---|
| 1995 | Rilutek® (riluzole) | Sanofi-Aventis | Slows the progression of ALS |
| 2017/2022 | Radicava® /Radicava ORS® (edaravone) | Mitsubishi Tanabe Pharma America | Intravenous (IV) infusion/Gel version May slow the loss of physical function |
| 2022 | Relyvrio (AMX0035) | Amylyx Pharmaceuticals | Removed from the market on 4/4/2024 due to clinical trial results |
| 2023 | Qalsody® (tofersen) | Biogen | Slows the rate of ALS progression for the approximately 2% of ALS patients who have an SOD1 gene mutation |
Clinical trials information on the new approvals:
Radicava (edaravone)
In the clinical trials, 137 participants were randomly assigned to receive either Radicava or a placebo. By Week 24, it was noted that individuals who received Radicava exhibited a smaller decline in their daily functioning compared to those who received the placebo.
Qalsody (tofersen)
The effectiveness of Qalsody was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical trial with 147 patients experiencing weakness due to ALS and a confirmed SOD-1 mutation verified by a central laboratory. Patients who received Qalsody showed statistically significant reductions in plasma NfL concentration at Week 28 compared to those in the placebo group. These findings indicate a potential clinical advantage for patients.
It is anticipated that AAN will revise their recommendations in the next year or two. Researchers are currently investigating the efficacy of newly approved drugs in conjunction with other medications and therapies. Sign up for alerts and stay informed on the latest published guidelines and articles.
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