Despite Limited Data, Relyvrio Offers a Glimpse of Hope for ALS Patients

When Tina Cascio was diagnosed with amyotrophic lateral sclerosis (ALS) in 2020, she knew what to expect. She had spent years as a caretaker, both as a nurse and for her mother, who died of ALS two years prior. 

Since her diagnosis, Cascio has taken every opportunity to participate in clinical trials for a shot at receiving an experimental treatment that could prolong her life. 

Relyvrio (sodium phenylbutyrate/taurursodiol), the first new FDA-approved ALS drug in five years, offers a glimmer of hope for patients like Cascio.

The drug approval has been met with skepticism because of insufficient clinical data and its sky-high price tag. But for ALS patients whose treatment options are limited, having even a slim chance of extending their lease on life may be better than nothing.

“All we’re asking for is a chance to try these drugs,” Cascio told Verywell. “If I just have six more months of watching the sun rise and set, that’s enough for me.”

More than 20,000 people currently live with ALS, with 5,000 individuals diagnosed each year. ALS is a neurodegenerative disease that weakens voluntary muscles, affecting patients’ abilities to speak, swallow, move, and breathe. In most cases, patients experience gradual paralysis and die from respiratory failure within three to five years after diagnosis. There’s currently no cure for the fatal disease.

Is Relyvrio Effective?

There are only three FDA-approved treatments for ALS. Relyvrio is the first drug to both extend survival for people living with ALS and to also slow the progression of the disease.

A small Phase 2 trial involving 137 people indicated that the drug slowed disease progression by about 25% over six months. In an extension of the trial, patients who received Relyvrio lived a median of 18 months longer than those who received a placebo. 

But in March, an independent panel advising the FDA concluded that the existing data didn't show "substantial evidence of effectiveness" for Relyvrio.

After a flood of patient pressure, the FDA changed course in September and approved the drug with the current data. Leaders in the ALS community said the potential benefit of the treatment is worth the uncertainty that it may not work.

“It’s not a cure, we understand that. But it is the first drug that both extends survival for people living with the disease, and slows the progression of the disease,” Andrea Pauls Backman, MBA, CEO of the Les Turner ALS Foundation, told Verywell. “There have been very few things in the ALS toolbox for neurologists to be able to offer people living with this disease.”

In the clinical trial, some patients experienced diarrhea, abdominal pain, nausea, and other common side effects. Jeffrey Rothstein, MD, PhD, Director of the Robert Packard Center for ALS Research at Johns Hopkins University oversaw the local trial for Relyvrio. He said the data shows no major safety concerns that would keep him from prescribing it.

If a patient’s life is prolonged thanks to Relyvrio, those extra months of life aren’t just “tacked on to the end,” when the patient’s autonomy and health is at its worst, Rothstein explained.

“If you would typically lose the ability to walk in a wheelchair at six months, you’re going to continue to walk until you make it to maybe nine months or 12 months. You’re just slowing that progress down,” he said.

That could allow patients to have a better quality of life in their last few years, Backman said.

“Everyone wants to remain as autonomous as they can in their own bodies. If you can feed yourself, you can tie your own shoes, you can get up out of bed, you can use the bathroom—if you can do these things on your own, that adds a tremendous amount to quality of life,” Backman said.

Scientists Await More Clinical Trial Data

About 80% of treatments for neurological disorders tested in Phase 2 trials aren’t found to be effective when tested again in a larger Phase 3 trial, Rothstein said. Scientists should know more about Relyvrio’s efficacy after the Phase 3 trial is completed in about a year.

The drug manufacturer, Amylyx, said that if the latest trial indicates that the drug does not actually perform well, the company would remove it from the market. But the company hasn’t said what it would consider to be a failure. And once the FDA approves a drug, it’s entirely up to the company to remove it from the market, unless there are major safety concerns.

In the meantime, Rothstein said Relyvrio is safe and that the potential that the drug works for some ALS patients is worth the risk that it doesn’t.

“What you’re getting for some patients is about a year or so of availability of a drug that has the potential to be effective,” Rothstein said. “Between now and when they finish the Phase 3 trial, there’s no question that I’m going to describe it for my patients. I see no reason not to.”

A Hefty Price Tag

Soon after the FDA’s approval, Amylyx received flack for the high cost of Relyvrio. A 28-day prescription of the drug is priced at $12,504, or about $158,000 per year before insurance.

Cascio, who has been dependent on disability benefits since her ALS diagnosis, said she “definitely cannot afford” the drug.

“We’re already fighting so hard for our lives. To have to fight with insurance companies and pharmaceutical companies on top of it—it’s exhausting. It truly is,” Cascio said. “There’s no reason to make money off of a terminal illness.”

In a conference call with investors, Chief Commercial Officer Margaret Olinger said the company would provide financial assistance to bring down costs for people with commercial insurance and provide it at no cost for uninsured and underinsured people who met certain criteria.

For many patients, the cost of the drug may be covered largely by their health insurance company.

An Exciting Time for ALS Research

Relyvrio is just one of many potential new advancements in ALS research brought on by mass social media attention to the disease. 

During the summer of 2014, videos of people dumping ice water over their heads dominated Facebook timelines worldwide. The viral social media challenge raised public awareness about ALS and brought in more than $115 million in the US and $220 million globally in about 6 weeks.

Most of those donations went to the ALS Association. More than $2 million went towards developing and testing Relyvrio.

The Les Turner ALS Foundation, Backman’s organization, used the funds it received to expand their laboratory research and support services for ALS patients.

In December 2021, President Joe Biden signed the Accelerating Access to Critical Therapies for ALS Act. The law sets aside $100 million per year towards ALS research and treatment development.

“We have done this work for many, many years. This is probably the most exciting time that we’ve seen,” Backman said. “These are huge, huge steps…They’ve built upon the foundation that existed for all these years, and now we’re seeing the fruition of this work.”

Correction - October 12, 2022: This article was updated to clarify the FDA advisory panel's decision in March and Amylyx's press statement.