FDA Approves ALS Drug Whose Study Was Partly Funded By Ice Bucket Challenge (cnn.com) 28
A new treatment for amyotrophic lateral sclerosis, or ALS, has been approved by the US Food and Drug Administration. CNN reports: The FDA announced approval of Relyvrio, developed by Amylyx Pharmaceuticals, on Thursday. The oral medication works as a standalone therapy or when added to other treatments, according to the company, and it has been shown to slow disease progression. Patients and some advocacy groups had urged the FDA to approve the drug, as there are limited treatments available for ALS, and the agency granted priority review in December.
In November, Amylyx submitted a drug application to the FDA for the medication, then called AMX0035, as an oral ALS treatment, seeking approval based on a Phase 2 trial that included 137 people with ALS who received either the drug or a placebo for 24 weeks. The study was funded in part by a grant from the ALS Ice Bucket Challenge, the viral social media campaign that started in 2014 involving people dumping buckets of ice water over themselves to raise awareness and money around ALS. The trial also showed that the drug was generally well-tolerated, but there was a greater frequency of gastrointestinal events in the group getting the medication. Amylyx is now continuing to study its safety and efficacy in a Phase 3 trial. In March, the Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 that a single Phase 2 trial did not establish the conclusion that the drug is effective in treating ALS.
One key difference between the FDA advisory committee's March and September meetings is that in the later meeting, Amylyx indicated that if the drug was approved but its Phase 3 trial results fail to confirm the drug's benefits, the company would consider withdrawing the drug from the market, Lynch said. She added, however, that the company didn't say specifically what it would view as a failure. "So at the vote, the advisory committee members switched, and most of them said, 'Yes, we are now convinced that this product should be approved.' And when they were asked why they changed their minds, some of them said, 'Well, the company said they would withdraw,'" she said. "And they were also convinced by patients' testimonies that they very much want to try this drug." But overall, the FDA's approval was based on Phase 2 trial data, which, Lynch said, may send a message to other pharmaceutical companies that they don't need robust Phase 3 trial data to get products on the market. Although people with ALS want access to this promising drug, there are concerns that such a message could open the door more broadly to the approval of medications that have not been proved to work, says Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania. "The FDA could later withdraw those products if needed, she said, but doing so without voluntary company agreement is 'a huge pain' and often requires a very lengthy process," reports CNN.
In November, Amylyx submitted a drug application to the FDA for the medication, then called AMX0035, as an oral ALS treatment, seeking approval based on a Phase 2 trial that included 137 people with ALS who received either the drug or a placebo for 24 weeks. The study was funded in part by a grant from the ALS Ice Bucket Challenge, the viral social media campaign that started in 2014 involving people dumping buckets of ice water over themselves to raise awareness and money around ALS. The trial also showed that the drug was generally well-tolerated, but there was a greater frequency of gastrointestinal events in the group getting the medication. Amylyx is now continuing to study its safety and efficacy in a Phase 3 trial. In March, the Peripheral and Central Nervous System Drugs Advisory Committee voted 6-4 that a single Phase 2 trial did not establish the conclusion that the drug is effective in treating ALS.
One key difference between the FDA advisory committee's March and September meetings is that in the later meeting, Amylyx indicated that if the drug was approved but its Phase 3 trial results fail to confirm the drug's benefits, the company would consider withdrawing the drug from the market, Lynch said. She added, however, that the company didn't say specifically what it would view as a failure. "So at the vote, the advisory committee members switched, and most of them said, 'Yes, we are now convinced that this product should be approved.' And when they were asked why they changed their minds, some of them said, 'Well, the company said they would withdraw,'" she said. "And they were also convinced by patients' testimonies that they very much want to try this drug." But overall, the FDA's approval was based on Phase 2 trial data, which, Lynch said, may send a message to other pharmaceutical companies that they don't need robust Phase 3 trial data to get products on the market. Although people with ALS want access to this promising drug, there are concerns that such a message could open the door more broadly to the approval of medications that have not been proved to work, says Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania. "The FDA could later withdraw those products if needed, she said, but doing so without voluntary company agreement is 'a huge pain' and often requires a very lengthy process," reports CNN.
Funding is not an issue .. (Score:3)
Now if this was to promote a treatment for hypothermia ...
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Or, it could be that all Fox News and right-wing media reports are entertaining political garbage sensationalized with fabricated evidence or context-washed cherry-picked facts?
Re: Funding is not an issue .. (Score:2)
This is the only one out of the 20 most recent stories on /. that links to CNN, so the premise of your question is already disproven. If CNN is paying for links on /. then /. is doing a pretty poor job of it.
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Nearly every Slashdot article these days
I assume you are using the phrase "nearly every" in an ironic way, or are really just this blatantly obvious a moron.
Awareness versus Action (Score:1)
This illustrates the difference between awareness and action. Like a lot of the woke stuff is mere awareness and virtue signaling, which does nothing if not only create backlash and resentment. Like right now, there is an awareness campaign for the women in Iran who are protesting having to wear the hijab, but nobody is doing anything meaningful to help them. Saying stupid stuff like "I stand with the women of Iran" is really dumb. You aren't standing with them, you are hiding behind a keyboard while they f
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Like right now, there is an awareness campaign for the women in Iran who are protesting having to wear the hijab, but nobody is doing anything meaningful to help them.
What do you want? Military action? An assassination plot?
The US is already imposing a long list of sanctions on Iran.
What action can a foreigner take?
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How about the action of respectfully letting protesters speak for themselves without attempting foreign interference in a sovereign nation? The protesters don't want foreign help, and the Iranian government has shown before how much it loves to highlight foreign support for protests as an excuse to legitimize crackdowns. So by shutting up you could at least do no harm.
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They are literally begging for foreign help.
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There are a lot of things we could be doing and should have been doing. For example:
First things we could have been doing: I don't know if it's Israel but it sure seems some organization(s), other than the CIA, have very deep penetration into their nuclear program. I mean it's clear Israel has been able to implement at least a Robert Hanssen / Aldrich Ames level compromise. I mean, IIRC, the strategy that used to be taught for these types of things is to have patience and get a large number of Manchurian ca
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No. For the love of $deity, please don't. At least consider the historic context before doing something stupid, ok?
1979 wasn't that long ago that nobody remembers it anymore. In the 1970s, the USA sent an incredible amount of military hardware into the Iran to build up the 4th largest army (and the probably 2nd most modern army at the time) on the planet. For the Shah, the enemy of the current regime.
Question for 100: What kind of propaganda tool could be forged out of the USA sending pretty much ANYTHING t
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Run a TOR server? (Score:2)
Like right now, there is an awareness campaign for the women in Iran who are protesting having to wear the hijab, but nobody is doing anything meaningful to help them.
What do you want? Military action? An assassination plot?
The US is already imposing a long list of sanctions on Iran.
What action can a foreigner take?
How about running a TOR server so that more people can avoid regime censorship?
(First read up on TOR requirements. Running a server using a slow, personal internet connection is apparently counterproductive. Lots of readers here have access to fast connections, or could maybe convince their companies to host a server.)
How about learning about social information management and helping out on social media? Respond to the negative press and soften it in some way ("mostly peaceful protestors"), respond to the r
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Re: Awareness versus Action (Score:2)
Poor turn of phrase (Score:3)
"The FDA could later withdraw those products if needed, she said, but doing so without voluntary company agreement is 'a huge pain' and often requires a very lengthy process,"
As much of a 'huge pain' as I'm sure the process is, I imagine most folks with ALS would argue that they can probably do you one or two better.
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Why are they blowing off the approval process? (Score:4, Interesting)
The approval process is supposed to be about analyzing research data, not social media fads.
Patents? (Score:2, Insightful)
I skimmed TFA (this is Slashdot, of course) but I could find no mention of this. This seems interesting information; I am going to be less interested in future fundraisers like the Ice bucket challenge, if it turns out that their profits go to the pockets of big pharma.
Re:Patents? (Score:4)
You really have to wonder about someone who finds someone making money more distasteful than someone dying a slow death of a horrible disease.
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OK, who is doing development of a patent free ALS drug? Nobody. That is why you can't donate towards it.
too bad (Score:2)
too bad the drug doesn't actually work.
Why not EUA? (Score:2)
Isn't this exactly what emergency use authorizations are for? It's a promising but unproven treatment for a deadly disease with no good existing treatments. An EUA lets patients who have little to lose start taking it, but doesn't undermine the legitimacy of the approval process. Why didn't they do that?