New Experimental Medication Offers Hope for Thousands of Americans Living With Lou Gehrig’s Disease

The Centers for Disease Control and Prevention estimates that 5,000 Americans are diagnosed with ALS (or amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease) every year. The debilitating and progressive illness robs individuals of their work life and career, causing muscle weakness; stiffness in the hands, feet, legs and ankles; difficulty walking and swallowing; muscle cramps; twitching; and slurred speech. Sadly, most people only live two to three years after being diagnosed with the illness.

While there is no known cure for ALS, a new treatment offers hope for those affected by the disease. The experimental drug, which is still in clinical trials, appears to slow the progression of the disease and improve the lifespan of those affected by the illness. 

Darryl C. Murphy of WBUR News, Boston’s NPR news station, has reported that the Food and Drug Administration is encouraged enough by a new treatment for ALS that they have “opened the door to early approval” for the medication.

“After completing two clinical trials for its drug AMX0035, the Cambridge-based company Amylyx reported earlier this year that the FDA wanted to see results from another round of clinical trials before the company could submit a new drug application, which is a proposal for a drug to be sold in the U.S.,” Murphy said.

However, that plan appears to be changing. 

“The agency typically requires three phases of trials before allowing a company to submit an application,” Murphy explained. “However, months later, after discussions with the agency and lobbying from patient advocacy groups, Amylyx announced its plan to submit the application in the coming months with the FDA’s blessing.”

In a statement praising the FDA’s decision, Amylyx co-founder Justin Klee said, “[I’m] giving them a lot of credit. They are finding the balance, at least in the case of our program, between ‘let’s make sure we have the right data, let’s make sure we run the right studies,’ but [also] understand the unmet need and time.”

Klee said the company intends to apply for approval of the new medication “in the coming months” and will also move ahead with conducting the final phase of clinical trials regardless of their application. He also said that Amylyx may also “create an expanded access program for those who wish to take the drug but do not meet the criteria for the trials.”

“[For] people with ALS, every single day matters,” Klee said. “We need to do all of these things to promote access as quickly and efficiently as we can.”

Encouraged by the preliminary results from the medication, health advocates are also pushing the FDA and Amylyx to move quickly with making the drug more widely available.

Brian Wallach, co-founder of the advocacy group “I AM ALS,” told Murphy that the company’s announcement “shows the power of the ALS advocacy community throughout the nation, making its voice heard and demanding the system do and be better.”

“It also shows what is possible when drug developers include patients in every step of the process,” Wallach said. “We will work relentlessly to make sure this is the first of many steps in changing the system to create more access to therapies for this 100% fatal disease.”


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