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DBL%20Hendrix%20small.png College chemistry, 1983

Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: derekb.lowe@gmail.com Twitter: Dereklowe

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June 22, 2010

Mylotarg and the FDA

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Posted by Derek

Someone completely outside the industry asked me the other day what I thought about the FDA. I replied that I had a lot of sympathy for them, actually. There's almost no way that they can avoid being yelled at by one group or another. You know - they're a bunch of foot-dragging nitpickers who are keeping effective medicines (things used in other industrialized countries, yet!) off the market here. Oh, hold it, it's Tuesday already - they're actually a bunch of incompetent industry shills who let all kinds of useless, toxic stuff through because they can't be bothered to do their jobs.

That's the sort of thing. If you want a good example of this, take a look at Mylotarg. Wyeth developed this oncology agent years ago for some forms of leukemia. It's a monoclonal antibody to CD33 (a cell-surface receptor found on leukocytes), conjugated to ozogamicin, a fairly aggressive chemotherapy agent. It was approved back in 2000 under "accelerated approval" rules, which are supposed to bring drugs for life-threatening conditions to market more quickly. The requirement is that companies continue to study such drugs after they're marketed, though.

Well, the studies have been completed on Mylotarg. It's not a very widely used drug, since it's only indicated for people 60 and older with particular forms of leukemia who aren't candidates for the more common therapies. But the numbers are in. . .and it turns out that people die more quickly while taking it than while taking the standard of care. Oh, dear. The drug has now been pulled from the market.

And there you have the FDA's dilemma: if they had sat on Mylotarg longer and required more studies, this probably wouldn't have happened. On the other hand, Wyeth might have decided to abandon it at that point - and not everything that gets accelerated approval is a Mylotarg. Some compounds that could actually help people could get lost that way. It's a real tightrope, and the rope is set up completely differently for every new drug. There's no way to get all these decisions right, but for life-threatening diseases, letting through more iffy compounds is still probably the right way to do it, I think.

Update: fixed all sorts of formatting and spelling issues, after taking a break from my real job to have a look (!)

Comments (10) + TrackBacks (0) | Category: Cancer | Regulatory Affairs


COMMENTS

1. Anonymoose on June 22, 2010 7:53 AM writes...

Not to be a nitpicker myself, but it's my-L-otarg, not myotarg. Doesn't really matter anymore, I guess...

Permalink to Comment

2. qetzal on June 22, 2010 8:22 AM writes...

Typo near the end of paragraph 3:

But the numbers are in. . .and it turns out that while taking it than while taking the standard of care.
Permalink to Comment

3. Sally on June 22, 2010 8:51 AM writes...

Actually, you could say that the FDA sat on this one a long time - a decade in fact, before deciding it was ineffective and more toxic than necessary. Good call by Pfizer to finally withdraw it, Wyeth should have done this several years ago.

Iressa was also approved by the same accelerated mechanism in 2003 and withdrawn much earlier due to lack of efficacy in the phase III trials. Not sure why this one took so long.

Permalink to Comment

4. Sally on June 22, 2010 8:54 AM writes...

Actually, you could say that the FDA sat on this one a long time - a decade in fact - before deciding it was ineffective and more toxic than necessary. Good call by Pfizer to finally withdraw it, Wyeth should have done this several years ago.

AZ's Iressa was also approved by the same accelerated mechanism in 2003 for lung cancer and withdrawn much earlier due to lack of efficacy in the phase III trials. Not sure why this one took so long.

Permalink to Comment

5. Sally on June 22, 2010 8:54 AM writes...

Actually, you could say that the FDA sat on this one a long time - a decade in fact, before deciding it was ineffective and more toxic than necessary. Good call by Pfizer to finally withdraw it, Wyeth should have done this several years ago.

Iressa was also approved by the same accelerated mechanism in 2003 and withdrawn much earlier due to lack of efficacy in the phase III trials. Not sure why this one took so long.

Permalink to Comment

6. Chemjobber on June 22, 2010 10:44 AM writes...

I think Derek was trying to link the Reuters article in my handle.

Permalink to Comment

7. CMCguy on June 22, 2010 2:07 PM writes...

The FDA is indeed in a tough position and does get blasted, often unfairly, from all sides. I can empathize with their dilemma to balance objectives with ever more scarce resources however know first hand dealing with them (or internal groups who play that role) can be frustrating because of both inflexibility to checkbox activities or inconsistencies in interpreting their own guidances.

This to me sounds like a case where the system in place worked as intended with accelerated approval meaning grant market authorization based on surrogate marker that was easier to study in clinical setting. The follow on data did not verify the expected clinical outcomes, and seems to show negative response which is truly sad. Was this a bad drug or a poor target/surrogate marker (or both)? Goes to illustrate how hard translation of science knowledge can be to medical applications. I suggest with out ability to use surrogates as possible routes to gain initial approval the drug development would be much more inhibited than already is.

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8. upset on November 27, 2010 8:30 PM writes...

I can't understand why, if it can help just a few people who have not other alternative, that it can't be avaliable to them. One year ago my husband was sent home, with hospice, after having aggressive threatment for CML. A few weeks later he was given mylotarg and his lekumia went into remission. Now he is to the point that he needs it again but it is not avaliable.

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9. Louise on October 2, 2011 4:30 AM writes...

It's Oct 2011 and my 50 year old neighbour has AML and has been put on Mylotarg. I thought it was out of favour and out of bounds since June last year, but this is England not the USA. Can anyone tell me if it is still in use here, legally, or if the ban is international?
Quick response would be greatly appreciated as he is, after a couple of weeks, getting jaundiced and gaining weight! He's an ex alcoholic by the way but at only 50 he seems too young to have been put on this chemo anyway.
Thanks folks.

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10. fudita drivers on September 11, 2013 7:59 AM writes...

I like this web blog its a master peace ! Glad I found this on google .

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