On April 27, 2022, the European Commission (EC) granted marketing authorization for Vydura® (rimegepant), a calcitonin gene-related peptide (CGRP) receptor antagonist, for both the acute treatment of migraine with or without aura and the prophylaxis of episodic migraine in adults with at least four migraine attacks per month. Rimegepant, an orally administered orally disintegrating tablet, is the first medicine authorized in the European Union (EU) for both the acute and prophylactic treatment of migraine. Migraine is one of the leading causes of disability worldwide, affecting approximately one in ten people in Europe alone. Globally, women are three to four times more likely to be affected by migraine than men.

The comprehensive clinical trial program has demonstrated the efficacy and safety of Vydura in both the acute and preventive treatment of migraine. Studies in acute migraine showed rapid and long-lasting relief of migraine headaches and other symptoms with a single dose, while the prevention study showed a significant reduction in migraine attacks with every-other-day administration.

The results of the phase 3 trial published in The Lancet showed that a single dose of rimegepant, compared to placebo, resulted in a greater reduction in migraine pain and associated symptoms after two hours. The prevention trial, also published in The Lancet, showed that rimegepant, taken every other day, resulted in a superior reduction in the number of migraine days per month compared to placebo during weeks 9 to 12 of the 12-week treatment period, a reduction that was maintained with continued use throughout the 12-month open-label extension period.

“This approval demonstrates the significant progress made in the acute treatment and prevention of migraines in recent years. The efficacy data and the favorable benefit-risk profile give hope that specialized care for sufferers who have not yet received sufficient help will be further improved,” commented Prof. Dr. Hartmut Göbel, specialist in neurology at the Kiel Pain Clinic.

The marketing authorization follows the recommendation for approval by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) from February 2022. The EU marketing authorization is valid for all 27 EU member states as well as Iceland, Liechtenstein, and Norway. Reimbursement authorization in Germany is still pending.

Vydura (rimegepant) targets a key component of migraine by temporarily blocking CGRP receptors. CGRP levels are elevated during a migraine attack, dilate blood vessels, and are involved in signal transmission via pain receptors. CGRP receptor antagonists work by reversibly blocking CGRP receptors, thereby inhibiting the biological activity of the body's own neurotransmitter CGRP.

The marketing authorization for Vydura was based, among other things, on the evaluation of the results of three Phase 3 studies for acute treatment, a long-term safety study for the acute treatment of migraine, and a Phase 3 study with a one-year open-label extension for the preventive treatment of migraine. Vydura is taken orally as needed, up to once daily to stop migraine attacks or every other day to prevent migraine attacks.

The most common adverse reaction in clinical trials with Vydura was nausea, occurring in 3% of patients compared to 1% in the placebo group, while hypersensitivity reactions, including rash, occurred in less than 1% of patients. Fewer than 2% of patients discontinued Vydura treatment due to adverse events. Vydura has no potential for dependence and was not associated with medication overuse headache or rebound headache in clinical trials, although overuse of any type of headache medication may worsen headaches.

At the beginning of this year, Pfizer and Biohaven entered into an agreement regarding the marketing of Vydura. Under this agreement, Pfizer holds the marketing rights for rimegepant in markets outside the US. Biohaven remains responsible for global research and development and retains the US market.

More than one billion people worldwide suffer from migraine. Migraine is characterized by debilitating attacks lasting four to 72 hours with a wide range of symptoms, including throbbing headaches of moderate to severe intensity, which may be accompanied by nausea or vomiting and/or sensitivity to sound (phonophobia) and light (photophobia). There is a significant unmet need for new treatments, as a substantial proportion of migraine sufferers are unable to work or function normally during an attack.