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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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August 14, 2012

Is Ampyra Any Good?

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Posted by Derek

I wrote here about Ampyra, the multiple sclerosis drug from Acorda Therapeutics, one that came close to the record for "simplest chemical matter in a marketed drug". (As it happens, Biogen Idec is making sure that it doesn't even have the title of "simplest drug for multiple sclerosis", and the shadow of valproic acid looms over this entire competition).

That post mentioned some doubts that had been expressed about how effective Ampyra is for its target: improving gait in MS patients. And now those doubts are increasing, because the company has been asked to conduct a trial of a lower 5 mg dose of the drug along with the approved 10 mg one (which was associated with seizures in some patients). And neither one of them met the primary endpoint. As that link shows, the company has several explanations - different endpoint than used before, higher placebo response than usual, wider variety of patients - but those are all ex post facto. Acorda wouldn't have set up the trial like this in the first place if they didn't think that the approved dose would work, and it didn't.

For a drug with a rather narrow symptomatic indication, that's not good news. And it comes as Acorda is still trying to get the compound approved in Europe. The cost/benefit ratio usually can't stand a big hit to the "benefit" term.

Comments (11) + TrackBacks (0) | Category: Clinical Trials | Regulatory Affairs | The Central Nervous System


COMMENTS

1. luysii on August 14, 2012 9:12 AM writes...

I can tell you, as someone who treated MS patients for decades, that almost anything done to them makes them feel better for a few weeks. This has been noted decades ago by Augustus Rose, an academic neurologist . The patients were for the most part intelligent middle class or upper (teachers, lawyers, MDs, etc.) people. Also, to be noted, is that 10 - 15% of the patients I saw were on Medicaid, so it wasn't an artifact of the patient mix.

Why the placebo effect should be so striking in MS isn't clear, but it appears to be a clinical fact.

Permalink to Comment

2. LJStewartTweet on August 14, 2012 6:52 PM writes...

I was struck by the many online videos of people reporting their own outcomes (Before and after) taking Ampyra (just Google Ampyra and MS for You Tube). The reported improvements in walking are sometimes amazing.

Does this one study mean that FDA would consider taking the Ampyra option away from patients?

I sure hope not.

Permalink to Comment

3. leonard on June 10, 2013 12:54 PM writes...

I TOOK AMPYRA FOR A MONTH. IT SEEMED TO WORK FOR TWO WEEKSW THEN (PROBABLY PLACEBO AFFVECT) THEN IT DID NOT WORK AT ALL .TOOK IT FOR REMAINING 3 MONTH AND HAD SIDE EFFECTS NEUROLOGIST SAID STOP . TOO BAD

Permalink to Comment

4. leonard on June 10, 2013 12:54 PM writes...

I TOOK AMPYRA FOR A MONTH. IT SEEMED TO WORK FOR TWO WEEKSW THEN (PROBABLY PLACEBO AFFVECT) THEN IT DID NOT WORK AT ALL .TOOK IT FOR REMAINING 3 MONTH AND HAD SIDE EFFECTS NEUROLOGIST SAID STOP . TOO BAD

Permalink to Comment

5. leonard on June 10, 2013 12:54 PM writes...

I TOOK AMPYRA FOR A MONTH. IT SEEMED TO WORK FOR TWO WEEKSW THEN (PROBABLY PLACEBO AFFVECT) THEN IT DID NOT WORK AT ALL .TOOK IT FOR REMAINING 3 MONTH AND HAD SIDE EFFECTS NEUROLOGIST SAID STOP . TOO BAD

Permalink to Comment

6. leonard on June 10, 2013 12:55 PM writes...

I TOOK AMPYRA FOR A MONTH. IT SEEMED TO WORK FOR TWO WEEKSW THEN (PROBABLY PLACEBO AFFVECT) THEN IT DID NOT WORK AT ALL .TOOK IT FOR REMAINING 3 MONTH AND HAD SIDE EFFECTS NEUROLOGIST SAID STOP . TOO BAD

Permalink to Comment

7. Dragan Spasovski on September 10, 2013 6:49 PM writes...

I am 53.MS was officially for me before 2 years. Although first symptoms are from 2004.
Now I am thinking to start with Ampyra .
But there are some dilemmas.
Is it possible to take it random?
Just at morning. And when it starts to affect to my walking for example .
What will happen if I take one week,after two days no , or similarly .
Is it possible to take for my birthday, a week before and week after. To celebrate a little .
When I take it few weeks, and stop, how many times it will still affect?
Where to find such instructions , to read answers
of practical questions ?

Best regards
Dragan Spasovski
Macedonia

Permalink to Comment

8. lilian weber on November 21, 2013 12:27 PM writes...

I took Ampyra. I loved it, tho I had resisted it. I began taking it in Oct. 2011. By summer 2012 I was in the hospital with a severe UTI that I had not recognised -- because Ampyra masked its effects. fortunately they mentioned that in the warning label, so my doctor new to treat for it right away. I continued taking Ampyra, tho I mentioned the UTI to one of Acadia's people who called in Aug. or Sept. Another such call came in Oct. or Nov., and the woman told me to stop taking it, which I did.
In Jan., 2013 I had a severe stroke. Any connection? Any similar cases? The warning label they have now about the seizures might have scared me off despite the benefits, but they didn't have it in 2012.

Permalink to Comment

9. Beth on March 8, 2014 11:58 PM writes...

I have been taking Ampyra for 3.5 years. Since starting it I have continued to get stronger. I have not had an MS attack since starting it. I am no longer as fatigued when taking it. For me, and I realize there are few of us, it has been the miracle drug. None of the other drugs helped me at all.

Permalink to Comment

10. Karen on March 15, 2014 4:30 PM writes...

AMPYRA has really worked well for me. It really makes the pain in my thighs get better and I can walk longer distances when I'm on it. My only complaint is that I get AMPYRA thru the assistance program and there is always a 3 - 4 mth gap at the first of every year while they reprocess my request for assistance. During this time my legs really ache all the time and my walking is greatly affected. I am very sensitive to dosing so their recommended dose works good for me. I wish it worked as well for others.

Permalink to Comment

11. Karen on March 15, 2014 4:30 PM writes...

AMPYRA has really worked well for me. It really makes the pain in my thighs get better and I can walk longer distances when I'm on it. My only complaint is that I get AMPYRA thru the assistance program and there is always a 3 - 4 mth gap at the first of every year while they reprocess my request for assistance. During this time my legs really ache all the time and my walking is greatly affected. I am very sensitive to dosing so their recommended dose works good for me. I wish it worked as well for others.

Permalink to Comment

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