Biohaven's Rimegepant for Migraines Heads to the FDA for Approval Following Successful Trial


Biohaven Pharma, based in New Haven, Connecticut, released positive data from its Phase III pivotal trial of rimegepant to treat migraine. The results were published in the New England Journal of Medicine (NEJM).

In the trial, 1,186 patients were either given a single dose of rimegepant or placebo. The co-primary endpoints were pain freedom and freedom from the symptom that bothers the patient the most at two hours after receiving the drug. The trial hit its co-primary endpoints.

“The results from this study demonstrate rimegepant’s potential as an effective treatment for migraine, a widespread, disabling disease that has seen only minimal improvements in acute treatment since the 1990s,” stated Richard B. Lipton, lead author, professor and vice chair of Neurology at the Albert Einstein College of Medicine and Montefiore Health System, director of the Montefiore Headache Center, and chair of Biohaven’s CGRP Scientific Advisory Board.

The patients in the trial, all adults, had at least a one-year history of migraine and two to eight migraine attacks of moderate or severe intensity per month. Of the 1,186 patients, 594 received the drug and 592 received placebo. Of those, 537 in the rimegepant group and 535 in the placebo group were evaluable for efficacy. The overall mean age of the patients was 40.6 years and 88.7% were women.

In the rimegepant cohort, 19.6% were pain-free two hours after receiving the drug, while 12% were pain-free in the placebo group. In the rimegepant group, 37.6% were free from their most bothersome symptoms two hours after dosing and 25.2% were in the placebo group.

The most common side effects were nausea and urinary tract infection.

It is estimated that 12% to 14% of all people, more than a billion worldwide, are affected by migraine headache. In addition to severe headache, symptoms may include nausea and sensitivity to light and sound. More than 75% of people who have migraine suffer at least one attack per month and more than 50% are severely impaired during them.

The most common treatments are triptan drugs, such as sumatriptan, eletriptan and rizatriptan, all of which hit the market in the 1990s. They stop and slow acute migraines by stimulating serotonin receptors, which reduces inflammation and constricts blood vessels. But not everybody responds well to them and because of the blood vessel constriction, shouldn’t be taken by individuals with cardiovascular disease or major cardiovascular disease risk factors.

Rimegepant is in a new class of drugs called gepants. Gepants’ method of action targets the receptors for the CGRP protein, which is associated with migraine. CGRP is released during an attack, resulting in pain. Gepants ease the pain and other symptoms by blocking the CGRP pathway.

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“These results confirm that rimegepant’s mechanism of action—blocking the CGRP pathway—effectively relieves pain and associated symptoms that occur during acute migraine attacks,” stated Lipton. “As someone who has studied CGRP blockers for more than a decade, I’m gratified to see their benefits confirmed in a large-scale clinical trial.”

Biohaven submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for rimegepant in the second quarter of this year.

Biohaven also launched a “Demand More” campaign that highlights what patients want and need from a migraine treatment. The program uses patients’ own words, which largely defines it as fast and reliable pain relief with the ability to return to normal life quickly without the need for repeated doses. They also, not surprisingly, want fewer side effects.

“Patients want more from acute treatment of migraine, and there is a growing demand and market for new options,” stated BJ Jones, Biohaven’s chief commercial officer, Migraine and Common Diseases. “We are committed to increasing awareness among physicians about how patients define real relief and what patients want and need from their acute treatment of migraine.”

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