Wednesday, 20 December 2017

MS News: too early for a high-dose biotin licence

The EMA has announced that Medday have withdrawn the licence application for high dose Biotin.

The EMA were not convinced by the data presented. 

The European Medicines Agency (EMA) has been examining data on high dose biotin (MD1003, Qizenday) since the application was accepted in September 2016. The EMA's Committee for Medicinal Products for Human Use (CHMP) concluded that the clinical data from two trials that enrolled 253 patients was not sufficient to assess the effectiveness or the safety of biotin. The company reserved the right to re-apply for licensing in the future.



Wc500240381 from BartsMSBlog


Trials are ongoing but as NDG has said "the data may have been less damning if the MRI atrophy data presented ECTRIMS had been positive".

The problem I see for this agent is they they don't really know how it works and this may be a fundamental problem. 

The trials have been designed to show that it an agent targeting progressive disease, but based on the early reports it suggested to me that the benefit was either symptomatic or perhaps repair and so the trial design may need to be very different. 

Why do I say this? 
It was because the reported benefits were occurring quickly.

So could this be another instance of Death of a Drug by Trial Design-meaning perhaps the drug could work but the trial was no designed so that the drug could shine.

20 comments:

  1. "So could this be another instance of Death of a Drug by Trial Design". Sorry to put barts on the spot. But are u saying there is therapeutic effect? Because I'm taking biotin. What Do barts advise? Can barts produce single page advise on supplements/diet/lifestyle about slowing ms progression? Even though there's No double blinded placebo controlled data? As scientists you would know better as opposed to patients experimenting themselves based on incomplete/dodgy small trials. For instance as relapsing patients, should I get my gp give me statins?

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    1. The EMA says the data is not strong enough, indicating there is no clear benefical effect, it does not say no effect. Medday are doing more trials and maybe that is what is needed. We would not be advising people to take these high doses without the evidence, it is currently not there. Likewise EMA have not approved is as safe.

      If the neuros would produce a page but there would be nothing on it that says doing this or that will slow progression.

      At present the jury is out on statins I understand there is a phase III trial so perhaps sign up for that.

      I am soory I have to be non-committal on such advice

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    2. Thanks MD. Completely understand. Problem is there's a lot of contradictory evidence on the net. Example for every research saying vitamin d3 is good for ms there's equally same number saying it has no effect. I guess short of a cure it's all just guess work.

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    3. And given the birth month and latitude data, any significant effect of vitamin D may be in utero.

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    4. Thanks MD2. Unfortunately I was born in hot hot country. Did me no good. Unless late onset is a consequences. What's the reasoning behind numbering MD, MD1, MD2? To convey rank? Would that make MD head chief after Dr Gavin?

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    5. Yeah it's rank, MD is the Big Chief Team Leader of the experimental arm of the team, the rest of us carry out his orders ;-)

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    6. HaHa but we don't issue orders, we are a really a cyborg collective with navigation.

      What MD2 is really saying is once we came up with the MD name which we did so we don't use names in case there is a crazy reading our posts on animals, MD2 was too lazy to come up with something else:-0
      I really mean it was such a good name, why use something else and MD2's thumbnail is way cool and was drawn by MD1.

      FYII...BCTL was invented by one of the post-docs for wayback whilst pulling MD's leg. We actually named a cell line we made BCTL-1.

      The classic name for a cell is FUJA.

      A person called JA had made a cell line but wouldn't give it away to other labs so another lab made the same thing and called it FUJA = F**K U JA.

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    7. BCTL and FUJA are brilliant names! Haha will be inscribed in research forever and readers will be none the wiser ;-)

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  2. Honestly, I do not understand the EMA here:

    1.) "...data on the medicine’s effectiveness were not robust enough.." -> Agreed, that's why SP2 ("Phase IV") is underway.

    Why was SPI (154 Patients / Phase-2) submitted as Phase 3 to start with? Phase-3 should have at least 200 Patients. Not?

    2.) "...that there were uncertainties regarding the medicine’s safety given the small number of patients treated with Qizenday.." -> Over 5.000 Patients in France for nearly 23 years...

    3.) "...more information was needed about how the medicine is absorbed, modified and removed from the body..." -> This is Vit. B7. Why is a better understanding of B7 within the Krebs-Cycle precondition for approval? There isn't even sufficient knowledge of some currently approved therapies.

    Or is this just a ploy to delay the process until more studies show better effectiveness resulting in higher list prices for the medication?

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    1. I have no idea....However I am guess the company had gone to EMA hoping to get approval...they haven't got it and so need to do more stuff.

      Phase VI = post marketing so as it is not approved yet it isn't phase IV.

      I know a lot of people in France have been getting it.

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    2. A ploy to get more money...I suspect every day the agent is not licenced it is costing the company thousands of euros

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    3. Re France, yep have a friend taking it reckons great :)
      although as you say, benefits reported very quickly, symptomatic?

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  3. Re:The problem I see for this agent is they they don't really know how it works and this may be a fundamental problem.

    Interferon beta 1a was never understood completely either. Also, I have to agree with Marc, this is vit B7, I would think the scientific community has plenty of data on its safety and bioavailability.

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    1. Copaxone too so now we can have a new mechansim every month.

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  4. Bummer. My neuro was going to put me in this trial beginning march. O well. up to newer and better. Perhaps it's for the best

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    1. The trial is ongoing and still recruiting. The EMA decision is simply saying we need more data; come back to us when you have more data. Not too dissimilar to oral Cladribine?

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  5. Does it matter if EMA approves or not. Pharmaceutical high grade biotin is already available. Just don't think it's effective. Otherwise ema would of approved it.

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    1. Guess your' right. Contacted my neuro about this, and seems we will just carry on with it

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  6. Ooooh can't wait. Another drug. Another year. As scientists you'd think your passion would show. My new year's resolution is to stop the self inflicted disappointed and stop coming here

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