SOMERVILLE, Mass. -The infamous Bruce Rosenblum knows the drug Relyvrio isn’t going to help him live. It could be useless or even have a negative impact.
However, after using a build-your-own version for several months but he couldn’t seem to purchase it before the start of summer, and is looking forward to getting it back up and running.
Late on Thursday on Thursday, on Thursday night, Food and Drug Administration approved the treatment for Relyvrio to treat amyotrophic lateral syndrome which is also called ALS. Rosenblum expressed his delight at the decision and has immediately asked for the prescription.
Early research suggests that the drug can be particularly beneficial at the beginning of the deadly disease which gradually deprives patients of their ability to control movements, and eventually, even breathing.
Rosenblum and others suffering from ALS do not have many alternatives.
There are only five other approved drugs for treating ALS The most recent was approved in 2017, however, none of them has been proven to reverse the irreparable decline in muscle strength. One drug has been proven in a few individuals to prolong their lives however only by three months, on average.
The evidence on the effectiveness of Relyvrio is in the early stages. A study that lasted six months in 137 patients, who half were given a placebo and the other half of them were taking the active drug found Relyvrio may slow declining rates. An analysis of the follow-up showed that patients taking the active drug lasted longer than those on the placebo.
A FDA advisory committee decided on March 1 that evidence was insufficient to justify approval. However, at another meeting earlier in the month, it changed its decision and suggested approval. This drug received approval in Canada at the end of June.
A larger study is expected to conclude next year. The company has said it will eliminate Relyvrio from the market in the event that the more thorough study shows that it doesn’t work.
“We can all agree that this drug is not going to stop the disease in its tracks,” Clive Svendsen is researcher at ALS research scientist in Cedars-Sinai located in Los Angeles, said before the FDA’s approval. Some drugs appear promising in small studies according to Svendsen who was not involved in the development of Relyvrio due to the fact that ALS is a progressive disease that occurs at different rates for various people.
Relyvrio is a mixture of two medications that are sodium phenylbutyrate as well as taurursodi it is believed to boost the energy of cells and stopping an event that could result in cell death. It is a pill that can be consumed either orally or through the feeding tube.
Common side effects are abdominal pain, diarrhea nausea, and upper respiratory tract infections However, the medication appears fairly innocuous.
What if Relyvrio be a “life-changing medication for those suffering from ALS?
Rosenblum who is 63, a computer software entrepreneur confirmed to have ALS in 2017 told me he’s open to trying everything that claims to slow his decline.
A study released in the past two years has brought Relyvrio in the eyes of numerous ALS patients. In the past, Rosenblum’s physician attempted to prescribe him sodium phenylbutyrate that was approved by the FDA for treating a different disease However, his insurance provider declined coverage.
Paying for a prescription through a specialist drugstore would have cost him $8400 monthly however, it was possible to locate a nearby compounding pharmacy which could do it for $1,050. “That I can stomach,” the pharmacist said. Taurursodiol is an affordable supplement.
He was taking the mixture daily since September of last year until in May when his prescribed ingredient suddenly became available. “Right now, I have no idea when I’m going to get it again,” Rosenblum stated. “I’ve used up all I had, as have most patients I know. We’re all waiting.”
Despite the lack of evidence to support efficacy, Rosenblum said the ALS patient population has been waiting to see Relyvrio’s approval, but frustrated by the advisory committee’s previous refusal to approve the medication. With the average patient living only 2 to five years after diagnosis, just a few months could have a major impact according to Rosenblum who lost two of his friends to the disease earlier this year.
Patients and advocates attended their participation at the FDA advisory committee meeting in early September, requesting the agency to allow Relyvrio because they are left with only a few options.
Vance Burghard told the committee that in 2017, he required assistance with pulling his pants up and couldn’t raise his arms up high enough to wear a T-shirt. He required a wheelchair to make it to doctor’s appointments and was forced to stop stocking shelves and assist customers in the shop which he was employed at.
At the end of 2018, following the medication, which was then referred to as AMX0035 the patient was no longer in need of wheelchairs and could travel with his wife in walking “many miles” through parts of Europe, China and Tibet.
“AMX0035 for me has been a life-changing drug,” Burghard stated, noting that his health is stable for the past three years. “I ask that you quickly approve it for the treatment of ALS so others afflicted with this disease can benefit from it.”
Patients require whatever assistance they can receive, experts have said.
“This is a great thing for our community. We haven’t seen (many) successes,” said Dr. Jinsy Andrews, director of clinical trials in neuromuscular disorders within Columbia University, and a participant in the Relyvrio trial. “Anything that has a good safety and tolerability profile and can buy any amount of time is acceptable for someone living with ALS.”