Axsome Therapeutics has finally jumped all the hurdles in a challenging regulatory saga, winning approval for its oral migraine drug Symbravo.
The rapid-acting treatment is approved for the acute treatment of migraine episodes regardless of whether they are accompanied by aura, which are the sensory disturbances—mostly visual—that can occur before and during an attack.
In clinical trials, Symbravo has been shown to provide patients with relief within two hours and to sustain its efficacy for up to 48 hours. Some patients can achieve these benchmarks with a single dose of treatment.
The company said it expects to make Symbravo available in approximately four months, but it did not divulge the drug's price.
Formerly known as AXS-07, Symbravo is a combination treatment of meloxicam and rizatriptan.
Meloxicam is a nonsteroidal, anti-inflammatory molecule developed with Axsome’s Molecular Solubility Enhanced Inclusion Complex (MoSEIC) rapid-absorption technology. It combines with rizatriptan, a 5-HT1B/1D agonist, which was previously sold by Merck as Maxalt before it went generic. Triptans are a first-line class of treatments for migraine.
“Results of multiple clinical trials demonstrate that Symbravo can provide rapid and long-lasting freedom from migraine pain, whether treatment is taken early in the attack while the pain is mild, or later in the attack when the pain may be severe,” Richard Lipton, M.D., professor of neurology and director of the Montefiore Headache Center at the Albert Einstein College of Medicine, said in a release.
Symbravo comes with a boxed warning for cardiovascular side effects, including the risk of heart attack and stroke. The FDA also warns of the potential of developing gastrointestinal side effects, such as ulcers.
The approval comes after the FDA rejected AXS-07 for manufacturing issues in 2022. Before that, Axsome’s regulatory timeline was impeded by a COVID-related inspection delay.
Even though the FDA didn’t require Axsome to run another trial, it took the company more than two years to address enough of the regulator's concerns to resubmit its application for approval.
The approval is based on results from three phase 3 trials. In the MOMENTUM trial, which tested the therapy in patients with moderate to severe migraine intensity, 77% of treated patients did not need rescue medication within 24 hours of dosing. Similarly, in the INTERCEPT trial, which tested the drug in patients when the initial pain was mild, 85% did not need rescue medication within 24 hours.
An estimated 39 million people in the U.S. suffer from migraines. Surveys of migraine sufferers found that more than 70% are not satisfied with their current treatment and “desire therapies that work faster, more consistently and result in less symptom recurrence,” according to Axsome's Jan. 30 press release.
New York City-based Axsome, which was established in 2012, is already on the rise thanks to Auvelity, which reached the market in 2022 for major depressive disorder and racked up sales of $291 million last year. With the approval of Symbravo, the company’s market cap has surged to $5.3 billion.
Analysts at Mizuho Securities wrote earlier this week that investors have tended to overlook Symbravo because of its “checkered regulatory path." Still, the analysts project Symbravo’s peak sales in the U.S. to reach $341 million in 2035, which comes up short of the company’s projection of between $500 million and $1 billion.
Axsome also is testing Symbravo in a phase 3 trial in patients who have inadequate responses to oral calcium gene-related peptide antagonists, which are the standard of care in the treatment of migraine.
Meanwhile, Axsome has two other pipeline assets—AXS-05 for Alzheimer’s disease and AXS-12 for narcolepsy—that have shown enough potential in phase 3 trials to set up regulatory filings this year.