FDA say no to alemtuzumab

F
Has the FDA got it right regarding alemtuzumab? #MSBlog #MSResearch


“For those of you who may missed this Genzyme press release, from the 30th December, concerning the FDA decision regarding alemtuzumab. The bottom line is MSers in Europe will have access to alemtuzumab and MSers in the US will not; unless they can afford to travel to Europe or Canada for treatment and pay for it privately.”



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Prof G

Professor of Neurology, Barts & The London. MS & Preventive Neurology thinker, blogger, runner, vegetable gardener, husband, father, cook and wine & food lover.

19 comments

  • Prof G,

    Have the FDA got it right? Was there sufficient blinding – were the people assessing the clinical outcomes blinded to treatments? Was there any issue with the blinding for treatment received (I assume impossible due to the methods of administration – should alemtuzumab patients been performing sham injections on themselves for two years?) Is there a significant issue with the lack of consideration of interferon NABS that would have made alemtuzumab look a lot better than it is?

    I would be interested, if only for my own sake as to the outcome of patients treated 10 to 20 years ago who presumably had severe disease to merit a novel unlicensed treatment.

    Will extension studies be done to make sure there is an idea as to what the long term treatment strategy after alemtuzumab should be 5, 10 or 15 years down the line so that patients don't end up in an evidence free zone at that stage? Relying on a relapse to prompt further treatment seems like a very crude measure.

    I speak as someone in limbo land after a CIS with many other subsequent symptoms that wouldn't add up to a relapse, but who finds them very debilitating who would have jumped at the chance of this drug early on.

    Many thanks.

    • The problem is the FDA are purists and don't accept secondary outcomes. The MRI data is objective and blinded and clearly shows that alemtuzumab is superior to interferon. The most compelling MRI data is the effect of alemtuzumab on brain atrophy, an integrator of end-organ damage, in year 2. Alemtuzumab takes the brain volume loss into the normal range.

  • Prof G,

    Happy New Year.

    As a layman I find it hard to understand why Canada, Australia and Europe have licensed this drug, but the FDA take a completely different view, particularly with regard to the trial design / validity of the results. Us in the UK aren't in the clear yet as NICE may well scupper this option for MSers.

    I've heard so many success stories about Alemtuzumab – putting RRMS patients into longterm remission. Yet the FDA seem to question whether the drug has any effect.

    • I am sure some of the London-based MSologists who do private practice would be willing to see you and offer you the treatment. I would recommend, however, this is done in collaboration with your US-based neurologist who is prepared to share in your care. The monthly monitoring will need to be done locally in the US.

      I assume a special funding package will need to be arranged with your insurance company to cover the costs of treatment outside the country and for the local monitoring.

  • I've commented on this on the Dec 'other' comments: A long time ago we were told on this blog – straight from the horse's mouth of Revd Coles – that we'd be provided with some info on the earliest Alemtuzumab treated RRMS cohort. For many of those who received it 'off label' outside the official trials, we're in the 15-20 year mark post-treatment. How these people are doing is off huge interest to the MS community – medical and patients. I appreciate it isn't trial data and therefore anecdotal but why is nothing been said about it? Prof G often talks about the 15 year Alemtuzumab 'experiment' to see if early treatment prevents SPMS – well, this kind of info would at least provide some insight into that experiment without waiting a further 15 years…

    Prof G – will you revert to Revd Coles, reminding him what he said on this blog, and ask for a response? Also, what is the length of treatment of your own earliest treated patients? How are they doing?

    I should add, I'm a big believer in Alemtuzumab and have myself been treated with it recently. However, I do think we're entitled to as much info as is out there. Otherwise, one assumes the info is negative and being withheld to protect Sanofi/Genzyme…

  • I agree that it is important that everyone sees the long-term data from alemtuzumab treatment.

    I promise you that Genzyme are not withholding it! In fact, they have no control over the data, since it comes from UK patients, treated from Cambridge.

    The reason that you have not seen these important results is simply because a scientific journal has not yet agreed to publish them. We have been trying for over a year to get the data published, but so far have been told that the information is not publishable, mainly because we do not have a satisfactory comparison group. We are working hard on this.

    I will let you know when the results are "out"

    Alasdair Coles

    • Yes, thank you very much for taking the time to reply.

      Is it incredible naive of me to ask why the info can't simply be made public, given the rejections to date? If it remains the case that no scientific journal will publish them does that mean they might never see the light of day? That doesn't seem right.

      Are you, at least, able to say whether this long-term data is positive, neutral or negative towards the concept of early treatment with Alemtuzumab being effective longer-term and potentially able to prevent SPMS? Unlike others on here who seem to delight in trying to knock each and every advance made in treating MS, I believe Alemtuzumab – early enough – represents a huge step forwards but, equally, would like to be able to base/revise my opinion on as much information as exists rather than on a partial view of the data.

  • My goodness, things are worse than I previously thought. American MSers, in a bizarre turn of events, want to flock to the UK for health care, and the ‘esteemed’ potency of Genzyme remains elusive because no “scientific journal has not yet agreed to publish them“. This is hardly the momentous breakthrough promised, is it?

    The FDA has thankfully questioned the efficacy of Campath-1H. That’s right: the world’s biggest and most pill-happy market has closed the door on Genzyme. What does that say about the state of things? While Don Giovannoni questions the prudent judgements of reputable health policy makers, we, the people, ask why no-one is convinced that alemtuzumab is the Holy Grail of MS (as suggested on this blog numerous times).

    The medical press is ignoring alemtuzumab, the world’s biggest health administration has rejected alemtuzumab, England’s NICE has been positively sceptical of alemtuzumab’s effectiveness, and now it transpires that some bloke probably sat somewhere in Delaware will fork out fortunes to come to Blighty in order to find some private Harley Street doctor who will administer alemtuzumab in return for big money. Come on, Don Giovannoni; you and I used to make fun of CCSVI lemmings that did that kind of stuff and now you’re actually advising something similar. What’s going on, bro?

    • Dre, it the way you say it that lets you get away with stuff. A comment yours would be routinely censored but because you turn your criticisms into a jokey tone, you get away with it.

      I think what you've said is valid, though hardly welcome. Although you sometimes bother me, I think yours is an important contribution in levelling opinions and provoking debate. Happy New Year, Dre.

    • Dre is right. Prof G lambasts the CCSVI practitioners for charging money for unendorsed treatments yet is advocating paying for something that has been also labelled as unconvincing by major health administrators. This is absolute hypocrisy on his part. Neither treatment has been certified as effective. He should have advised whoever to wait until the drug gets a green light in their country rather than saying British private neurologists will probably treat them for money.

    • I think you are missing the point. Lemtrada has been licensed in both Canada and Europe. There is no reason why an American can't be prescribed the drug in these countries. In contrast Liberation therapy for CCSVI has not been licensed in any country.

    • Anon 8:41, that's no defence, Canada has even licensed CCSVI! Lemtrada may have been approved by Europe but it is not available in any country there.

    • The FDA issue is based around trial design. Ask ANY neuro who has been involved in prescribing Alemtuzumab – whether part of a trial or off-label – and they will tell you it is an incredibly effective drug for RRMS. The trial data also is extremely persuasive even if one ignores the comparison with Rebif (which is where the FDA's issues lies). It completely stops all disease activity (MRI and clinical) for a substantial number of people. It reduces annual relapse rate to about 0.1 – that's 1 relapse every 10 years in a previously quite active population. Brain magnetisation transfer ratio is returned to pretty much normal (a marker for atrophy). New lesions are extremely low. Is it the panacea for all of MS's potential evils – no, of course not. But it's extremely effective – as good as a cure, so far, for some people who have taken it – and as good as we have right now. The FDA also over-state the risks based on its use for other conditions at much higher levels – AND fail to understand just how tough life with untreated or inadequately treated MS can be. To compare Alemtuzumab and CCSVI is, frankly, ridiculous.

    • I believe you can get Lemtrada prescribed already in few EU (richer) countries like Germany, Denmark, Finland…

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