Remember the "Ice Bucket Challenge" in 2014? All around the world, people were dumping buckets full of ice water on their heads and committing to donating money in support of a good cause.
The challenge was started by two people with amyotrophic lateral sclerosis, more commonly referred to as "ALS" or Lou Gehrig's disease, and helped raise awareness for the neurological condition.
And what you may not know is that the social media trend also raised $115 million in donations for the ALS Association.
The association invested some of the donation money into the development and trial of a drug for ALS, called Relyvrio, according to its press release.
This year, Relyvrio was approved by the Food and Drug Administration for treatment of ALS.
"We thank the millions of people who donated, participated, and enabled us to invest in promising therapies like AMX0035 [Relyvrio] that will immediately help people living with ALS," Calaneet Balas, president and CEO of the ALS Association, said in the statement.
ALS is a rare nervous system disease that impacts the nerve cells that control voluntary muscle movement, according to the National Institute of Neurological Disorders and Stroke.
In most patients, symptoms get worse over time, causing respiratory failure that leads to death within three to five years of the onset of the disease, according to Eufrosina Young, board-certified neurologist and ALS specialist at the Upstate University Hospital's department of neurology.
Relyvrio, in combination with other ALS treatments, can add nearly seven months onto patients' lives based on data collected from its trial, says Young. Patients are already calling her office, asking if they can add the drug to their treatment, she says.
"Risk for first hospitalization was measured at a 44% decrease in those that were receiving the drug," she notes, "These are very significant markers of disease progression."
But while people living with ALS are excited and hopeful about the drug's approval, some experts are nervous that it was greenlit too soon and without enough scrutiny.
"There's just a general concern out there that maybe the trial is wrong," David Rind, chief medical officer for the Institute for Clinical and Economic Review, told NPR.
For experts like Rind, some of the hesitancy can be attributed to the fact that FDA approval of Relyvrio was made after just one study of only 137 patients.
Normally, for a drug to become FDA-approved, two smaller independent studies, with less than 1,000 patients, showing similar results or one large study with positive results across the board are required.
"Given the serious and life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this instance," the agency wrote in its benefit-risk assessment.
And while Young would've preferred that the trial had more participants, she does agree with the FDA's decision.
"Across the board, everybody wants a bigger trial, and everybody wants more evidence," she says, "But I think at the end of the day we can all agree, too, that a majority of ALS patients do not have that time."
Another trial with more participants is in the works to further test the drug's efficiency, and Young is hopeful that it will provide more assurance.
Until the trial is completed, she plans to only recommend Relyvrio as a combination therapy, in conjunction with pre-existing medications.
Yet, another potential issue to consider is access to the drug itself. After Relyvrio's approval, Amylyx, the drug's manufacturer, announced that it would cost $158,000 a year.
"We are working to ensure that people with government-funded insurance, like Medicare or Medicaid, will have access to RELYVRIO as quickly as possible," Amylyx said in a statement to CNBC Make It.
"For U.S. residents with ALS that are uninsured or underinsured, meet certain financial eligibility criteria, and who have exhausted all other options, we intend to provide RELYVRIO at no cost."
A spokesperson from Amylyx said the company plans to use the money from sales to create new treatments in the future, according to Futurism.
"The main battle after the FDA's granted approval is the battle with medical insurance to cover the cost. Even with medical insurance, patients may or may not have a copay," Young says.
"These patients have terminal disease and deserve to receive this medical treatment."