Migraine sufferers who cannot get relief from existing medications may soon have a novel treatment option, a new trial suggests.
The study, of nearly 1,700 patients, found that a pill called ubrogepant worked better than a placebo pill at halting migraines in progress.
The drug has not yet been approved by the U.S. Food and Drug Administration. But it belongs to a new class of medications called CGRP inhibitors that has come to the market in the past year.
CGRP is a small protein released by the trigeminal nerve during migraine attacks. It's believed to play a key role in generating migraine misery, explained lead researcher Dr. Richard Lipton, who directs the Montefiore Headache Center at Albert Einstein Medical College in New York City.
The three approved CGRP inhibitors are all injection drugs that are used regularly, to prevent migraine attacks.
Ubrogepant is different because it's a tablet that treats migraines in progress. Another oral "gepant," called rimegepant, is also in the pipeline. Data on both drugs have been submitted to the FDA for approval, according to the companies developing them.
In the new study, ubrogepant worked better than a placebo at easing pain and other migraine symptoms, such as nausea and sensitivity to light or sound.
Of patients who used the real drug to treat a migraine attack, 22% of those on a higher dose were pain-free within two hours. That compared with 14% of the placebo group. Similarly, 39% of ubrogepant users were free of their "most bothersome" symptom within two hours, versus 27% of placebo users.
The study, funded by drug's maker, Allergan, is published in the Nov. 19 issue of the Journal of the American Medical Association.
According to Lipton, the new gepants could make a "big difference" for certain migraine patients.
They include people who do not get relief from current acute treatments, and those who cannot take the medications because of side effects or safety concerns, Lipton said.
Right now, medications called triptans are the standard treatment for more severe migraine attacks. The drugs, which came out in the 1990s, stop migraines by stimulating receptors for the brain chemical serotonin, which reduces inflammation and constricts blood vessels.
But not everybody responds to the medications. And because of the blood vessel constriction, people at high risk of heart attack or stroke cannot take them.
Triptans also have side effects - like numbness, dizziness and sleepiness - that can make them difficult to take.
Gepants work through a "novel mechanism," Lipton said, which means they might help some patients who do not respond to triptans. And they do not constrict blood vessels.
Lipton has financial ties to both Allergan and Biohaven Pharmaceuticals, maker of rimegepant.
It will be "exciting" to have new options for patients who cannot take triptans, said a neurologist who was not involved in the study.
"There haven't been any new acute treatments in a long time," said Dr. Rachel Colman, of Mount Sinai's Icahn School of Medicine in New York City.
Questions do remain, Colman pointed out. The latest trial tested the effects of only a single treatment, and it's not clear how consistent patients' responses will be over time, she said.
Long-term safety and side effects are also unknowns - though, Colman said, "so far, the tolerability data looks good."
Nausea was the most commonly reported side effect, affecting 2% of ubrogepant patients within two days of taking the drug.
In the United States alone, more than 37 million people suffer from migraines, according to the American Migraine Foundation. People with milder migraines may find relief with general pain relievers like naproxen and acetaminophen.
But for some migraine sufferers, Colman noted, "there's a significant need" for new tactics.
According to Lipton, Allergan said it expects FDA approval as early as next month.
Sources: Richard Lipton, M.D., professor and vice-chair, neurology, and director, Montefiore Headache Center, Albert Einstein College of Medicine, Bronx, N.Y.; Rachel Colman, M.D., assistant professor, neurology, and director, low-pressure headache program, Icahn School of Medicine at Mount Sinai, New York City; Nov. 19, 2019, Journal of the American Medical Association.