ALS patients and families across the country are applauding the FDA for fast-tracking a drug that could slow the decline of physical function by 33 percent.
The drug is called Radicava and it is the first new treatment approved specifically for ALS in 22 years.
The drug’s list price is $1,000 per infusion, which amounts to $146,000 a year.
KREM 2 set out to verify the following:
At that cost, will most patients be able to access the new drug?
What does Radicava do?
Is this a breakthrough?
WHAT IS ALS:
ALS is infamous for the way it strips a person of their movement and voice. It leaves the mind acutely aware of the shackles of the disease.
"ALS is a really bad disease,” said Dr. Greg Carter from St. Luke’s Rehabilitation Center. “It causes some of the highest disease burden of any disease I've ever seen."
He said he has seen a lot of inquiries recently about Radicava.
HOW RADICAVA WORKS:
Radicava is also known as Edaravone and is not a new drug. In fact, it was approved in 2001 in Japan to treat stroke. In a six month trial of 137 people with ALS, half were given infusions of Radicava and half were given a placebo.
After six months, experts said, the people receiving Radicava slowed the decline of physical function by an average of 33 percent.
“We’re really excited for this really historic approval,” said Tom Larson, from M.T. Pharma America, the company behind Radicava.
Larson said the FDA did not put any limitations on when ALS patients should or should not take the drug, but agreed it will not restore a patient’s movement after it is lost. Meaning, giving it early to help preserve function will be key.
"Once the nerve cells are gone, the nerve cells are gone,” said Dr. Travis Denton, a chemist at WSU College of Pharmacy.
Denton is working on his own drug to combat ALS, but is watching Radicava closely.
The drug essentially helps protect brain cells from an onslaught of free radicals brought about by disease. When those free radicals start winning the war, the ALS patient loses movement.
"Everyday what do I say? Antioxidants, antioxidants, antioxidants,” explained Denton. “That's really what this molecule is. It's an antioxidant that can get into your brain. And that's a really tough nut to crack."
To put it plainly, the cost of Radicava is high. Infusions are $1,000 per treatment and a full year of treatment can add up to $146,000.
“One of the things that was a really important goal is to make and ensure Radicava is accessible to people with ALS,” said Larson.
“We plan to have a very generous program where we plan to remove as best we can the barriers to access as it relates to out of pocket expenses,” added Randy McGonigal, the M.T. Pharama America director of market.
McGonigal said they have a program called “Searchlight Support” that will help patients access the drug. In some cases, he said, uninsured or under-insured patients may not be charged at all.
Given all the variables, McGonigal said he can’t say exactly how much the average person will pay out of pocket.
“It's hard to say, here's exactly how we are going to help every single patient, because you're right, it varies so wildly,” McGonigal said.
ALS advocacy organization Team Gleason said while Radicava is not a cure, it is a step in the right direction.
"The data shows Radicava can slow the progression in those recently diagnosed. Although the affect is said to be modest, it is more than was previously available. And for people with ALS, that’s incredible news,” a spokesperson wrote in a release.
"What does it mean to the average patient?” asked Denton. “I think if you can afford it, I think you should take it."
Dr. Greg Carter, St. Luke’s Rehabilitation Center
Dr. Travis Denton, chemist at WSU College of Pharmacy
M.T. Pharma America