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New Hope For ALS Patients After FDA Reverses Course On Possible Treatment

The Food & Drug Administration (FDA) campus in Silver Spring, Md.  This photo was taken Oct. 14, 2015. (Andrew Harnik/AP)
The Food & Drug Administration (FDA) campus in Silver Spring, Md. This photo was taken Oct. 14, 2015. (Andrew Harnik/AP)

The Food and Drug Administration has opened the door to early approval for a new ALS drug treatment despite limited, though promising, evidence of its effectiveness.

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.

The agency typically requires three phases of trials before allowing a company to submit an application. 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.

Amylyx co-founder Justin Klee praised the agency for having a sense of urgency toward ALS treatment.

“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 understand the unmet need and time,” said Klee.

The company plans to submit the application in the coming months and will still conduct the final phase of clinical trials. While awaiting the FDA reviewKlee said 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. We need to do all of these things to promote access as quickly and efficiently as we can,” Klee said.

ALS stands for amyotrophic lateral sclerosis. It’s a devastating disease that causes the nerve cells involved with muscle function to stop working and die. As a result, patients progressively lose control of their muscles, experience muscle weakening and then paralysis

According to the federal Centers for Disease Control, most people with ALS live just three to five years after symptoms develop, but results from clinical trials for Amylyx's treatment showed it can slow the disease's progression.

Such results moved ALS advocates to pressure the FDA and Amylyx to quickly make the drug more widely available.

The ALS Association even called out the federal agency for approving a controversial Alzheimer’s medication, aducanumab, in June despite unconvincing evidence that the drug improves lives for patients, while AMX0035 received different treatment.

In a statement released earlier this week Brian Wallach, co-founder of another advocacy group, I AM ALS, said Amylyx’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,” he added. “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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Darryl C. Murphy Twitter Reporter
Darryl C. Murphy is a business reporter at WBUR.

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