FDA Makes Multiple Decisions, Vemircopan Misses Primary End Point, AAN 2026: Top Interviews

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at the top stories in Neurology.

Beginning with some FDA news, the administration was busy during the week of May 4, 2026, making a number of decisions on potential new therapeutic agents, including granting a couple of approvals, and extending the review of a supplemental biologics license application (sBLA). The NeurologyLive^® team has been hard at work covering all the agency movements to make sure you are up to date. Our coverage includes the latest FDA approvals, new designations, submissions, resubmissions, and clinical trial initiations and holds.

The first piece of FDA news in the first week of May was a successful Type C meeting with Clene Nanoscience, who is now motioning toward an Accelerated Approval NDA Filing for their drug CNM-Au8 in ALS.¹

May 8th was a busy day for the FDA with 3 separate decisions. First, the FDA approved a sBLA for efgartigimod alfa-fcab (Vyvgart; argenx), expanding its indication to include adults with acetylcholine receptor antibody (AChR-Ab) seronegative generalized myasthenia gravis (gMG) who do not have detectable AChR-Ab.² Then, the FDA approved intravenous ocrelizumab (Ocrevus; Genentech) for the treatment of relapsing-remitting multiple sclerosis (RRMS) in pediatric patients aged 10 years and older who weigh at least 55 pounds, expanding a high-efficacy anti-CD20 therapy into a younger population.³ Finally, the FDA extended its review of Eisai and Biogen’s sBLA for a once-weekly subcutaneous autoinjector starting regimen of lecanemab-irmb (Leqembi Iqlik) in early Alzheimer disease (AD), pushing the Prescription Drug User Fee Act (PDUFA) action date to August 24, 2026.⁴

In other news, final results from a double-blind, multicenter, phase 2 randomized clinical trial (NCT05218096) showed that treatment with vemircopan, an oral factor D inhibitor, did not significantly improve clinical outcomes compared with placebo in adults with acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR-Ab+ gMG), leading investigators to terminate the study early after the prespecified efficacy threshold was not met.⁵

Despite evidence of pharmacologic target engagement and alternative pathway inhibition, no statistically significant differences were observed across primary or secondary efficacy measures. The findings, published in JAMA Neurology, highlighted ongoing challenges in developing complement-targeted therapies for gMG beyond terminal complement component 5 (C5) inhibition, while raising questions about the therapeutic potential of selectively targeting the complement alternative pathway (AP) in this disease setting.⁵

The 2026 American Academy of Neurology (AAN) Annual Meeting, held April 18-22, in Chicago, Illinois, brought together thousands of clinicians, researchers, trainees, and industry leaders for one of the field’s premier neurology conferences. This year’s meeting featured plenary sessions focused on ongoing controversies in neurology, presentations highlighting advances in translational science, late-breaking clinical data, award lectures, and discussions spanning nearly every subspecialty in neurology.

Throughout the meeting, NeurologyLive^® provided onsite coverage from the convention floor, capturing key scientific updates and conversations shaping the future of neurologic care. In addition to reporting on emerging data and presentations, the team conducted a multitude of interviews with experts across the field, from early-career investigators and leading clinicians to notable science educators such as Bill Nye. 

As coverage from the meeting winds down, the NeurologyLive team looked back on some of the standout interviews and discussions from AAN 2026. Featured interviews included Rodolfo Savica, MD, PhD, of Mayo Clinic Rochester, Michael Levy, MD, PhD, of Mass. Genreal Hospital, Edith Graham, MD, of Northwestern Feinberg School of Medicine, and more!

To read the full piece and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. After successful FDA meeting, Clene plans accelerated approval NDA submission for CNM-Au8 in ALS. Clene Nanoscience. News release. May 4, 2026. Accessed May 11, 2026. https://invest.clene.com/news-releases/news-release-details/after-successful-fda-meeting-clene-filing-accelerated-approval
2. Argenx Announces U.S. FDA Approval Expanding VYVGART and VYVGART Hytrulo for Use in All Adult Patients Living with gMG. Argenx. News Release. May 8, 2026. Accessed: May 11, 2026. https://argenx.com/news/2026/press-release-3291372
3. FDA approves Ocrevus (ocrelizumab) for pediatric relapsing multiple sclerosis. News release. Genentech. May 8, 2026. Accessed May 11, 2026. https://www.gene.com/media/statements/ps_050826
4. Update on FDA Priority Review of LEQEMBI® IQLIK™ (lecanemab-irmb) Subcutaneous Injection as a Starting Dose for Early Alzheimer’s Disease. News release. Eisai and Biogen. May 8, 2026. Accessed May 11, 2026. https://investors.biogen.com/news-releases/news-release-details/update-fda-priority-review-leqembir-iqliktm-lecanemab-irmb
5. Saccà F, Gialdini G, Howard JF, et al. Efficacy and Safety of Vemircopan in Generalized Myasthenia Gravis: A Randomized Clinical Trial. JAMA Neurol. 2026. doi:10.1001/jamaneurol.2026.090