An experimental treatment for ALS is one step closer to getting the green light, and patients with the disease say it's a major step forward. An FDA Advisory Committee voted in favor of approving a drug that could help slow the progression of the disease.
Steve Kowalski has been living with ALS for the last five years.
"I was sent home after my diagnosis with really little hope that there was some treatment available that would help," he said.
Now that the FDA Advisory Committee voted to support approval of an experimental drug, he has some hope. The manufacturer, Amylyx, said a clinical trial showed the drug "significantly slowed" the rate of functional decline from ALS compared with people given an placebo. The company said the drug was generally well-tolerated. It said "gastrointestinal events" happened more often in the people who got the medication. Patients like Kowalski are urging the full FDA for approval.
"For those folks who are on the bedsides as we speak getting delivered this devastating news, this is some hope that will help them in their process of dealing with the horrible diagnosis,” he said.
ALS is also known as Lou Gehrig's Disease. It affects as many as 30,000 people in the U.S. It's a disease that causes muscles to weaken, and eventually affects patients’ ability to talk, swallow, move and breathe. The full FDA is expected to make a decision by the end of September. If approved, Kowalski said all eyes will be on the insurance approval process.
"Which is yet another hurdle that I think many folks are going to have to deal with, to fight to get approval to have the medication covered," he said.
