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Promising New Migraine Drugs in Development

Promising New Migraine Drugs in Development

The first medications developed specifically for migraine prevention in more than 50 years show promise in phase II drug trials. Both drugs target CGRP, a protein long thought to have a significant role in in migraine attacks (See Ellen’s excellent explanation of CGRP to learn more). By blocking CGRP, the drugs stop migraines from starting in the first place.

Here’s a summary of the two studies presented at this week’s American Academy of Neurology meeting1:

Study I

Participants: 163 people who had five to 14 days of migraine attacks each month

How the drug was tested: Participants received either a single IV dose of the drug, ALD403, or a placebo. Participants were followed over 24 weeks.


  • After 24 weeks, participants who received the drug had an average of 5.6 fewer migraine days a month (a 66% reduction)
  • 16% who received the drug were migraine-free after 12 weeks
  • 61 of patients had their number of migraine days cut in half
  • 33% of patients had their number of migraine days cut by 75%
  • Those who received the placebo had 4.6 fewer days per month (a 52% decrease) after 24 weeks
  • No one who received the placebo was migraine-free

Side effects: Side effects were the same for both groups.

Study II

Participants: 217 people who had four to 14 migraine days per month.

How the drug was tested: Over 12 weeks, participants received an injection every two weeks. Some received the active drug, LY2951742, while others got a placebo.


  • After 12 weeks, those who received the drug had an average of 4.2 fewer migraine days a month (a 63% decrease)
  • Those who received the placebo had 3 fewer migraine days a month (a 42% reduction) after 12 weeks

Side effects: Those who received the drug were more likely to have side effects than those who received the placebo. Side effects included pain at the injection site, upper respiratory tract infections and abdominal pain. Overall, though, the drug was found to be safe and well-tolerated.

Those are definitely good early results. More, larger studies are needed to confirm the findings and determine the best delivery method for the drugs. (They cannot be taken orally. Ideally, patients would be able to inject it themselves once a month.) If you’re interested in joining a clinical trial, search for ALD403, AMG 334, LBR-101 and/or LY2951742 on These are the “code names” for the drugs that are in development.

You may remember reports of Telecagepant, another experimental drug that targeted CGRP, which was abandoned after phase III trials. Drug development was halted after studies found potential liver toxicity. Even though the drugs currently being developed also target CGRP, they work in a different way. Drugs like Telecagepant are known as small-molecule CGRP receptor antagonists. The new drugs are large-molecule antibodies, which avoid the issue of liver toxicity because they are not broken down in the liver. (An interesting aside: the small-molecule drugs were not good for daily use because of liver toxicity, but they are 1/15 as toxic to the liver as triptans. They are now being studied for use as migraine abortives.)2

This new research is quite exciting for migraineurs and researchers are also hopeful about the findings. The lead authors of the studies said:

“These results may potentially represent a new era in preventive therapy for migraine.” –Peter Goadsby, MD, PhD, UC San Francisco

“We’re cautiously optimistic that a new era of mechanism-based migraine prevention is beginning.” –David Dodick, MD, Mayo Clinic Arizona

The drug could be available as soon as three years from now. Are your fingers as tightly crossed as mine are?

This article represents the opinions, thoughts, and experiences of the author; none of this content has been paid for by any advertiser. The team does not recommend or endorse any products or treatments discussed herein. Learn more about how we maintain editorial integrity here.

  1. "New Drugs Offer Hope for Migraine Prevention." American Academy of Neurology (AAN). 22 April 2014. <>.
  2. "Could an Antibody Prevent Migraine?" Neurology Reviews. June 2013; 21(6):1,30. <>


  • Ellen H
    5 years ago

    What about those of us who are Deficient in Liver Enzymes? My doctors have long known that I cannot metabolize meds. The meds either do not work, or they
    build up in my system producing some weird and painful adverse effects: loss of memory, pain in my back, insomnia, flu like symptoms, suicidal thoughts, and the list goes on. What are the chances that someone might create Liver Enzymes so that the rest of us can metabolize the medications and find some relief. I personally have lived with migraines my entire life and have taken numerous meds and would really like to have the necessary enzymes to metabolize meds so maybe I could lead a slightly more productive life.

  • Kristenlucas
    5 years ago

    I am currently on this study and just started open label this week. I have had no side effects yet. During the double blind I was getting 3 injections of varying amounts. I have been in the study since March 2014. I have had a reduction of migraines during the double blind portion of the study, but won’t know whether I was getting placebo or drug until next year. (The study has 3 options, placebo, low dose or high dose drug).

  • Vicki
    6 years ago

    As always, thank you for sending along this info. At least we know they are working on us, right??

  • marlenerossman
    6 years ago

    And how many years to get FDA approval? And how long after that until the drugs hit the pharmacy shelves? Don’t hold your breath waiting for these…

  • bluebird
    6 years ago

    I am encouraged to hear that some new strategies might become available since the old ones have not worked well for me. Thank you for reporting on this “hot off the press” at the professional meeting. I am tapering off Topomax and have just gotten enough thinking and emotional energy back to feel some enthusiasm about life…even with chronic daily migraine…But the prospect of some new approach gives me more hope.

  • mrst53
    6 years ago

    I now have kidney problems and am concerned about new drugs and how they affect my kidneys and my liver. So now, I have to be careful about what I have to take for my migraines. Ooh to get old…..

  • scotchbonnet
    6 years ago

    Hi mrst53, sorry to hear you now have kidney and liver problems, if migraines weren’t enough to contend with, were they because of medication you have taken for prevention of migraines, ie, Topimax, or Epilim.

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