Amylyx filing in Canada

[Nikki J]

Amylyx Pharmaceuticals Plans Regulatory Filing of AMX0035 for the Treatment of ALS in Canada

Amylyx Pharmaceuticals plans to submit a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in the first half of 2021.

www.businesswire.com

 

[Bestfriends14]

I was just going to post this very thing. This looks promising, though I'm scared to get my hopes up. Wayne and I have been following this drug's success with the community for quite some time and have been impressed thus far. I hope it can be pushed through quickly.

I'd be interested to hear other members opinion on what they think of this, as well.

 

[GXTrex]

As per the doctors recommendation, my PALS is taking TUDCA off of amazon as this medication is very hard to get. Has there been any research done comparing AMX0035 vs TUDCA? If this is approved in the US I assume it would be worth it to switch to AMX0035.

 

[Nikki J]

There is some research on tudca alone though nothing of the scale of the amylyx study. I think everyone believes the combination is better. Your dad could achieve the same thing though by getting a prescription for sodium phenylbutyrate. There is discussion on other threads about this with a link to the journal article some neurologists are using to support their appeal. We still don’t know whether we will get approval or a phase 3

 

[jethro]

combination? i think that PB is added just as scavanger. tudca is a main drug/component. pb is expensive, tudca not. how to get money from a cheap drug if you can buy it in a supplement store?? why nobody tried pb before? enturage/synergy? aha...
tudca showed some results in trial (25% slower progression), but that was not reliable, cause cohorts were small and period was 1 year. currently, there is a trial just with tudca in eu. planned 440 pals. they enrolled half. it is very had to collect 440 pals in different countries. results will be in 2023.

 

[Nikki J]

They have different targets so the combination is a 2 pronged approach. There was a phenylbutyrate study in 2009 and it showed some improvement in a biomarker. It was only 26 people and the fda wanted more toxicity studies and they couldn’t get money to fund that So it was abandoned Phase 2 study of sodium phenylbutyrate in ALS - PubMed

 

[jethro]

9 to 21g/day vs 3 x 2g/day. ok, it was safety and that is ok. most interesting thing is
"Histone acetylation was decreased by approximately 50% in blood buffy-coat specimens at screening and was significantly increased after NaPB administration. Blood levels of NaPB and the primary metabolite, phenylacetate, increased with dosage. While the majority of subjects tolerated higher dosages of NaPB, the lowest dose (9 g/day), was therapeutically efficient in improving histone acetylation levels."

 

[Samkl]

How are the 2 components combined and taken in Amx0035? Were they compounded as one substance or 2 separate solutions, and does it matter. If PALS did manage to get both, how is it best to take them?

 

[KateMMA]

This is great news; hopefully it will get approved quickly so it's available to more PALS soon.

I wonder if Health Canada is more likely to approve it without a phase 3 trial than the FDA. The article says it was being submitted after "feedback from Health Canada" so I would think that means they have already given some indication that it would be approved without a phase 3 trial.

Is anyone familiar with the Health Canada process for drug approval?

 

[ShiftKicker]

Here's a quick overview:

The Drug Review and Approval Process in Canada – An eGuide -

Download information about The Drug Review and Approval Process in Canada - an eGuide.

spharm-inc.com

This process is separate from reviewing a drug for coverage by Provincial health agencies.

 

[jethro]

@Samkl - both are powders. i dont know how they applied 4 grams for one serving. 3 grams pb and 1 gram tudca. i bought tudca via alibaba. if i get somehow pb, i'll pack mixture into capsules. tudca has an awful taste, even i lost sence of taste. tried to put it in a smootie - no chance. i had to put it in a capsule. it slows deterioration app 25%. trial was 6 months. i think that better results will be in a longer period.
note: tudca is not udca, but tudca=taurursodiol.
i'm in.
--
here is a part of a trial:
Results: A total of 177 persons with ALS were screened for eligibility, and 137 were randomly assigned to receive sodium phenylbutyrate-taurursodiol (89 participants) or placebo (48 participants). In a modified intention-to-treat analysis, the mean rate of change in the ALSFRS-R score was -1.24 points per month with the active drug and -1.66 points per month with placebo (difference, 0.42 points per month; 95% confidence interval, 0.03 to 0.81; P = 0.03). Secondary outcomes did not differ significantly between the two groups. Adverse events with the active drug were mainly gastrointestinal.
Conclusions: Sodium phenylbutyrate-taurursodiol resulted in slower functional decline than placebo as measured by the ALSFRS-R score over a period of 24 weeks. Secondary outcomes were not significantly different between the two groups. Longer and larger trials are necessary to evaluate the efficacy and safety of sodium phenylbutyrate-taurursodiol in persons with ALS. (Funded by Amylyx Pharmaceuticals and others; CENTAUR ClinicalTrials.gov number, NCT03127514.

 

[Samkl]

For anyone in Australia - tudca powder is available from at least one, (and probably more) supplement maker. I’ve been getting tudca online from the US viaAmazon, but will investigate this source now. I don’t know where they get it from.

 

[lgelb]

In terms of the timing, Amylyx has targeted submission by June. With priority review, there could be a decision by the end of the year.

 

[KateMMA]

Thanks for the information shiftkicker. It looks like a relatively quick turnaround.

Igelb, it would be great if they can decide that soon and maybe have it available next year.

Fingers crossed!

 

[Mon]

Hi all.
I found the Amylynx news very interesting, especially since i am a resident of Ontario. I read the study and results. Please can someone help me interpret the data. Did it show extended life by 6.5 months for those who got the drug vs. Placebo? I know all participants had bulber plus 2 other regions involved. Is the hope here that if this medication os given to others in a earlier stage that it prolongs life by more than 6.5months and slows progression?