The FDA has approved the use of 75-mg rimegepant (Nurtec ODT; Biohaven) in the acute treatment of migraine in adult patients, according to Biohaven, marking the company’s first agency approval of a product.1
Approval of the oral antagonist of the calcitonin gene-related peptide (CGRP) receptor was granted based on the results of 2 clinical trials: the phase 3 Study 303 and the long-term, open-label safety trial, Study 201. Rimegepant achieved statistical significance on the co-primary endpoints of pain freedom and freedom from the most bothersome symptom (MBS) at 2 hours post-dose compared to placebo. Additionally, the treatment demonstrated statistical superiority at 1 hour for pain relief and return to normal function.
“The FDA approval of Nurtec ODT marks an important milestone for the migraine community and a transformative event for Biohaven,” Vlad Coric, MD, CEO of Biohaven, said in a statement. “Millions of people suffering from migraine are often not satisfied with their current acute treatment, at times having to make significant tradeoffs because of troublesome side effects and reduced ability to function. Nurtec ODT is an important new oral acute treatment for migraine that offers patients the potential to quickly reduce and eliminate pain and get back to their lives.”
In its clinical development, more than 3100 patients were treated with more than 113,000 doses of the therapy. Overall, rimegepant was generally well tolerated; the most common adverse event (AE) was nausea, which occurred in 2% of patients who received therapy compared to 0.4% of patients who received placebo.
In data presented at the 2019 American Academy of Neurology Annual Meeting of Study 303
Freedom from pain at 2 hours posttreatment was achieved for 21.2% of patients in the ODT rimegepant arm compared with 10.9% of those in the placebo group (P <.0001). Moreover, the patient-reported most bothersome symptom (MBS) at 2 hours was significantly reduced in the rimegepant group (35.1% vs 26.8%; P = .0009).2
Pain relief was achieved at 60 minutes for 36.8% of patients in the ODT arm compared with 31.2% in the placebo group (P = 0.314). At 90 minutes, half of the patients in the rimegepant arm experienced pain relief (49.6%) compared with 37.2% in the placebo arm (P <.0001). Pain relief at 2 hours was 59.3% versus 43.3%, for rimegepant and placebo, respectively (P <.0001).
“I see many patients in my practice whose lives are disrupted by migraine, afraid to go about everyday life in case of a migraine attack,” Peter Goadsby, MD, PhD, professor of neurology, director King's Clinical Research Facility, King's College Hospital, said in a statement. “Many feel unsure if their acute treatment will work and if they can manage the side effects. With the FDA approval of Nurtec ODT, there is renewed hope for people living with migraine that they can get back to living their lives without fear of the next attack."
In Study 303, sustained pain relief was achieved for 47.8% and 27.7% of patients for 2-24 hours and for 42.2% and 25.2% for 2-48 hours, in the rimegepant and placebo groups, respectively (P <.0001). Sustained freedom from MBS was achieved for 27.1% and 17.7% for 2-24 hours and for 23.2% and 16.4% for 2-48 hours, for rimegepant and placebo groups, respectively (P <.01).
The ability to function normally returned for 38.1% of patients at 2 hours in the rimegepant arm compared with 25.8% in the placebo group. Moreover, there was a lower probability of requiring rescue medications in the rimegepant arm compared with placebo (14.2% vs 29.2%).
"We believe Nurtec ODT will be the first of many innovative Biohaven medicines to become available to treat devastating neurological diseases, a therapeutic category many other companies have abandoned,” Coric added. “We are dedicated to helping patients with these conditions, who often have limited or no treatment options, live better, more productive lives."
In January, data published which included 2 patients with migraine experiencing suboptimal response to medication suggested that concomitant use of rimegepant and erenumab (Aimovig; Amgen), an FDA-approved preventive for migraine, can effectively treat refractory migraine. The work, conducted by Kathleen Mullin, MD, medical director, clinical research, New England Institute for Clinical Research, and colleagues, is the first clinical report documenting that 2 CGRP therapies can be used this way.3
Although those data are an exciting first step, Mullin and colleagues noted that “the mechanism underlying the benefits of concomitant use of a small molecule CGRP receptor antagonist and an anti-CGRP receptor antibody is unknown and requires further study.”
1. Biohaven's NURTEC™ ODT (rimegepant) Receives FDA Approval for the Acute Treatment of Migraine in Adults [press release]. New Haven, CT: Biohaven; Published February 27, 2020. Accessed February 27, 2020. prnewswire.com/news-releases/biohavens-nurtec-odt-rimegepant-receives-fda-approval-for-the-acute-treatment-of-migraine-in-adults-301013021.html.
2. Lipton RB, Coric V, Stock EG, et al. Efficacy, Safety, and Tolerability of Rimegepant 75 mg Orally Dissolving Tablet for the Acute Treatment of Migraine: A Phase 3 Double-Blind, Randomized, Placebo-Controlled Trial (Study 303). Presented at: 2019 American Academy of Neurology Annual Meeting. May 4-10, 2019; Philadelphia, PA. Poster 075.
3. Mullin K, Kudrow D, Croop R, et al. Potential for treatment benefit of small molecule CGRP receptor antagonist plus monoclonal antibody in migraine therapy. Neurology. 2020;00:1-5. doi:10.1212/WNL.0000000000008944