The town of Sommerville, Massachusetts.
It's no secret that Bruce Rosenblum understands the medicine Relyvrio won't save his life. It's possible that it won't even make a difference. But he had been using a homemade version for a while until early this summer when he found out he couldn't obtain any more, and now he's keen to get back into it.
The Food and Drug Administration (FDA) granted Relyvrio approval late Thursday for use in the treatment of amyotrophic lateral sclerosis (ALS). Rosenblum exclaimed, "This is fantastic news!" and promptly asked for a prescription. Preliminary results from the drug's clinical trials suggest it may be most effective in treating the earliest stages of the lethal condition.
When faced with ALS, Rosenblum and others like them are left with few choices. Only five other medications have been approved to treat ALS (the most recent in 2017), and none of them have been found to slow or halt the progressive weakness and paralysis that characterises the disease. Only one has been found to lengthen life in some patients, and even then, only by an average of three months.
Could Relyvrio truly be a 'game-changer' for patients with ALS?
A software entrepreneur named Rosenblum, 63, who was diagnosed with ALS in 2017, has said he is open to trying anything that would slow the disease's progression. The attention of many people with ALS was drawn to Relyvrio thanks to a study published two years ago. Sodium phenylbutyrate had already been approved by the FDA to treat another rare illness, but Rosenblum's insurance company refused to pay for it. By himself, it would have cost $8,400 per month at a specialty pharmacy, but he discovered a local compounding pharmacy that made it for $1,050. I can handle it, he said. Taurursodiol is a low-priced supplement you may get today.
Regular use of the combo began in September of last year and continued until May of this year, when he discovered that the prescription substance was suddenly unavailable. Rosenblum expressed his uncertainty about when he will be able to get the item again. "I, like many other sufferers I've met, have depleted my reserves. Everyone is holding their breath while they wait."
Rosenblum stated that the ALS patient community has been eager for Relyvrio's approval and dissatisfied by the advisory committee's earlier rejection of the treatment due to the lack of proof for benefit. Following losing two friends to the disease last summer, Rosenblum knows firsthand how even a few months may make a tremendous impact in the average patient's survival time of 2 to 5 years after diagnosis. At a hearing before an FDA advisory group in early September, patients and advocates urged the agency to approve Relyvrio because it is one of very few options for them.
When can I get Relyvrio and how much will it cost?
Relyvrio's manufacturer, Cambridge, Massachusetts' Amylyx Pharmaceuticals, has vowed to have the drug available to the public within around six weeks, the same time span after it was approved in Canada. Prior to the approval, co-CEO Justin Klee stated that multiple medications, including Relyvrio, will be used to delay the progression of the condition. He remarked that because "ALS is a complicated, multifaceted illness," several strategies will be required to treat it.
Klee and his co-CEO, Josh Cohen, have transformed their scientific epiphany into a full-fledged mission. When working on a problem as devastating as ALS, Klee says, "it's impossible not to feel enthusiastic every day, but also feel like we're not doing enough." "We feel a tremendous obligation to use this platform for positive change now that it is within our reach. We can only hope this is the beginning.... We believe there is strong evidence in favour of our treatment, but it is not a panacea. Our efforts must be sustained."
Board member Larry Falivena, who was diagnosed with ALS in 2017, stated in an emailed statement, "The ALS community has proved that our advocacy can effect decisions that are being made concerning our health." The company has invested $2.2 million on Amylyx's early research and stands to earn $3.3 million from medicine sales.
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