ALS drug will be pulled from US market after study showed patients didn't benefit

[BumRushDaShow]

Source: ABC News/AP

April 4, 2024, 10:05 AM


WASHINGTON -- The maker of a drug for Lou Gehrig’s disease that recently failed in a large study said Thursday it will pull the medicine from the market, acknowledging it didn't help patients with the deadly neurological condition.

Amylyx Pharmaceuticals announced it will voluntarily halt sales and marketing of the drug in the U.S. and Canada, where new patients will no longer be able to get a prescription.

“While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS," company co-founders said in a statement. Patients already taking the therapy who wish to continue will be able to enroll in a program to receive it for free.

The Food and Drug Administration approved the much-debated drug, Relyvrio, in September 2022, following a years-long advocacy campaign by patients with amyotrophic lateral sclerosis, or ALS. The drug's failure is a bitter disappointment for patients and advocates, who have pressed the FDA and other federal agencies to fund and approve more experimental therapies for the fatal muscle-wasting disease.

Read more: https://abcnews.go.com/Business/wireStory/als-drug-pulled-us-market-after-study-showed-108849403

[OAITW r.2.0]

1. My heart goes out to those afflicted with ALS.

My mom was diagnosed and died from it within 18 months. I can't think of a more horrible disease to contract,

[4lbs]

2. If it didn't really work, why was it allowed in the first place?

Oh wait, you mean a drug manufacturer falsified results to get their snake oil approved? Color me shocked!

Or was it all those people afflicted with the disease who cried about getting it approved without any solid data to back it up?

So.... will the drug maker also refund the monies paid for the drug since it went to market?

[BumRushDaShow]

3. "If it didn't really work, why was it allowed in the first place?"

From the article excerpt -

The drug's failure is a bitter disappointment for patients and advocates, who have pressed the FDA and other federal agencies to fund and approve more experimental therapies for the fatal muscle-wasting disease.

When you have various organizations pushing for "something", then it can get approved for what is called an "Investigational New Drug". I expect the ALS Foundation may have been one of those advocate groups pushing for it.

[4lbs]

4. Yeah, unfortunately, that seems to be a case of globbing onto anything

in a vain, desperate attempt at hope.

I mean, come on, if it takes 5 years, then it takes 5 years. Don't try to rush it and have everything in 1 year. It will often lead to disaster, like this.

I'd rather the FDA take its time to do things right. Get the science and the data to back it up, properly.

I'm sensing Alzheimer's and Parkinson's may wind up like this.

[BumRushDaShow]

5. This happened with Laetrile

https://www.webmd.com/cancer/amygdalin-cancer-treatment

[4lbs]

6. Yeah, reminds me of all those people who have cancer, then end up spending

their life savings on wild, pie-in-the-sky, "treatments" that don't work, but promise great results, in another country.

Alternative crap that doesn't do anything but drain your bank account and make some fake "doctor" richer.

Steve Jobs died this way.

[karynnj]

10. Jobs relying on alternative medicine was even sadder than people with no real

Successful medicines. The reason there is early compassionate use before full studies is because people with things like ALS have no better choice. This time the new drug failed, but what if an unproven drug was successful? It could mean life or death for someone getting it early.

[4lbs]

11. "successful" based on what scientific measure?

Otherwise, I would simply state SSS (Small Sample Size).

To get a larger sample that proves the drug in question actually works in general, requires thousands of positive results.

You can always then claim, this drug "works", under this specific set of circumstances. Mmmm-kay, but what if you don't fall into that set? Well... then.. it won't work. Because the science and data don't support it working.

I'd rather it be proven to work under all circumstances, not just one or two. Yeah, that might take an extra 5 or 10 years, but hey, sometimes, the approval process needs to be slow and take its time, to make sure everything is in order, and the drug in question works for all cases, not just one or two, that you don't qualify for anyway.

[karynnj]

12. The article says that it failed.

Why do your think that it was seen to "fail" due to small sample size? In general, the sample size narrows the confidence interval. There is no reason to think that a drug that fails to show a positive result in a study with a small sample would show a positive impact if a bigger study is done.

From the article, the FDA and the drug company bent over backwards to give the drug a chance based on less proof because of the seriousness of the disease and the fact that there were few alternatives.

[CountAllVotes]

7. Five years is nothing to study a drug

I am amazed the way they are pushing these various drugs through the FDA without the proper research.

Time and research both are required.

I find this sickening!

The patients have no real say in this matter while they are the ones that are hoping for a cure.

[4lbs]

8. Yeah, true. I want the FDA to take at least 5 years to get "approval" for a drug.

The drug may have been in development and in clinical trials for about 10 years before that, costing hundreds of millions.

But, when the FDA gets the initial results and such, take a long time to glean the results and data to make sure everything is good. That could take at least 5 years.

People point at the COVID vaccine and how it was done in less than a year with FDA "emergency use approval". That's because the science and technology behind it was around 20 years old. The original COVID vaccine was developed from 2002, based on initial musings from 1999. The mRNA technology was decades old. They merely had to put in a "new engine" (new mRNA strain for the latest COVID-19 viruses) and test it over 6 months to make sure it was still working properly.

That is a far cry from developing a new drug from scratch. That takes 10 years or more at the minimum. 5 to 6 years to develop and do trials. 5+ years for a proper study from the FDA.

[catbyte]

9. I can understand trying anything when faced with this diagnosis. It's a guaranteed death sentence.

It's not like cancer where you have a fighting chance, no matter how slim. There is no hope whatsoever. When my dad was diagnosed with ALS, I think I would've tried anything on the off-chance that something, anything might work. It would've been worth a shot. And while I usually agree with the 5-year testing rule for most drugs, when presented with a diagnosis that pretty much assures you that you'll be dead within 3 years, why not try it? There's really nothing to lose. It's not like a new blood pressure or diabetes med.

My dad was always a no-nonsense kind of guy and he died 6 months after he was diagnosed. But, as I said, I would've tried anything. You can't imagine what a diagnosis like that is like until it happens to you or a loved one.