A new class of migraine medicines could make a "big difference" to those in desperate need of help.
The US Food and Drug Administration has not yet approved a drug for drug use, but extensive clinical testing suggests that this oral pill could work safely where other treatments do not.
Within two hours of taking it, the researchers found that ubrogepant could stop severe migraines in its wake, acting significantly better than placebo and with less risk than other drugs.
"Ubrogepant, as a potential new drug for acute migraine treatment, will provide much-needed innovation for the disease that is wasting time for millions of people," says neurologist Richard Lipton, a consultant for Allergan, a test-sponsoring pharmaceutical company.
Exhaustive migraine is more than just a bad headache, and it is much harder for doctors to treat. In the US, more than 38 million people suffer from this neurological disease, and surveys show that less than a third are satisfied with their care.
When it comes to effective and safe treatment, each patient is a little different, and in some severe cases the options are limited and can even make things worse.
Since the 1990s, triptans have continued to be the most popular class of migraine medicines in cases where over-the-counter drugs simply do not work.
But while triptans can help reduce pain and inflammation, they also narrow the blood vessels in the body. As a result, these drugs are not safe for people at high risk of heart disease or stroke, and others are not responsive at all.
Despite this, there have been no new treatments for acute migraines for a long time. In fact, the FDA has just recently approved a new class of medicines, called cheetahs, that can help stop severe headaches before they even begin.
Unlike tryptans, which target serotonin in the brain, cheetahs use monoclonal antibodies to target a molecule called the calcitonin gene-related peptide (CGRP), which has a known role in migraines.
So far, only three CGRP inhibitors have been approved by the FDA and all are injections. If ubrogepant is considered safe and effective enough, it will be one of the first oral cheetahs that can prevent acute migraines.
In a phase 3 clinical trial, which was random, double-blind, and placebo-controlled, researchers tested two different doses of ubrogepant on 1,686 patients, all of whom reported migraines between 2 and 8 times per month.
Participants were given either a tablet of ubrogepant containing 50 mg, 25 mg, or a placebo. During the test, they were instructed to take one tablet as soon as possible, or within 4 hours of moderate or severe migraine.
If that initial dose was not sufficient to stop the pain, a second dose was allowed and this was randomly selected so that the patient either received a placebo or repeated dose of ubrogepant. "Rescue drugs", such as acetaminophen, NSAIDs, opioids, anti-emetics or triptans, were only administered when both doses did not work.
Of all those taking the lower and higher dose of ubrogepant, over 20 percent are pain free within two hours. In comparison, placebo was only down 14 percent.
Getting rid of the most excruciating symptoms was a little more difficult and required a higher dose of ubrogepant. In this case, only those taking 50 mg were significantly better than those taking placebo.
"Current results show that 50 mg of ubrogepant has the potential to address key treatment goals in the acute treatment of migraine," Lipton and colleagues write.
"The ubrogepantin mechanism of action may make it an option for people who are not responding to currently available drugs."
It is an effective drug, but not as impressive as other drugs already on the market. Previous research, for example, has shown that triptans can show up to 70 percent of patients with safe and effective response rates within an hour.
Neurologist Stephen Silberstein, who was not included in the study, told CNN that although ubrogeptants may be useful to those who do not tolerate triptans, they are no better and are not a magic cure.
Instead, it is better to consider the urogepant a new promising treatment for those patients who have entered the fissures.
"For the first time in a long time, we have gone from news to lots of good news," Silberstein wrote in a recent survey review.
"We have switched to these new medicines for the acute treatment of migraine and we have new medicines for the prevention of migraine."
More research is needed to evaluate the long-term safety of these drugs, but according to Lipton, FDA approval could arrive as early as next month.
This study was published in PIT.