Neurology News Network: Top FDA Approvals of 2021

WATCH TIME: 4 minutes

Welcome to this special edition of Neurology News Network. As we wrap up 2021, we decided to look at 3 of the top FDA approvals from this past year.

The FDA approved Biogen's investigational anti-amyloid, disease-modifying agent aducanumab for the treatment of Alzheimer disease, marking it as the first novel approval for the neurodegenerative condition since 2003. The agent has been given the trade name Aduhelm.The therapy was approved under the Accelerated Approval pathway, which will require Biogen to conduct post-approval phase 4 studies to confirm the benefit of the drug. The agency noted that in the case that the anticipated clinical benefit is not confirmed, it "has regulatory procedures in place that could lead to removing the drug from the market." In its decision, the agency noted that the late-stage development program consisted of 2 trials, 1 which met its primary end point and 1 that did not. It noted, however, that in all of the studies in which it was evaluated, data suggested aducanumab "consistently and very convincingly" reduced amyloid plaques in both a dose- and time-dependent fashion.

The FDA approved 75-mg rimegepant (Nurtec ODT; Biohaven) for the preventive treatment of migraine, Biohaven has announced, adding to its prior indication for the acute treatment of the headache disorder, and marking it as the first oral calcitonin gene-related peptide (CGRP) antagonist to be approved for prevention and the first to be approved for both acute and preventive therapy.The orally disintegrating anti-CGRP tablet was originally approved in a 75-mg dose for the acute treatment of migraine in February 2020—the first approval of a therapy for Biohaven.2 The application for the preventive indication was accepted for review by the agency in October 2020.The decision was made based on supporting data included in the supplement new drug application (sNDA) from the pivotal phase 3 clinical trial, known as Study 305. Those data showed that the CGRP therapy decreased monthly migraine days by 4.3 day per month at month 3, compared to the placebo group’s 3.5-day reduction.

The FDA approved ponesimod (Ponvory; Janssen Pharmaceutical), an oral S1P1 modulator that functionally inhibits S1P activity and reduces circulating lymphocytes, for the treatment of adults with relapsing multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. The FDA decision was made after assessing supporting data from the phase 3 OPTIMUM study, a head-to-head evaluation that examined the safety and efficacy of 20-mg ponesimod against 14-mg teriflunomide (Aubagio; Sanofi) in adults with relapsing MS. Those data were previously presented at the 2019 ECTRIMS annual meeting and showed a 30.5% greater reduction in annualized relapse rate (ARR) with treatment with ponesimod compared with teriflunomide at week 108, with respective rates of 0.202 and 0.290, respectively (P = .003). The secondary end points—which consisted of fatigue-related symptoms as assessed with the Fatigue Symptom and Impact Questionnaire-Relapsing Multiple Sclerosis (FSIQ-RMS), an MS-specific, 20-item patient-reported outcome measure—were also positive in OPTIMUM.

For more direct access to expert insight, head to NeurologyLive.com. This has been Neurology News Network looking forward to a great 2022. Thanks for watching.