FDA Action Update, Week of May 4, 2026: Approvals, Extended Review, and Plans for NDA Filing

The FDA was busy early this month, 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).

With all the treatments that have progressed through the pipeline of clinical development, the NeurologyLive^® team has been hard at work covering all the agency movements to make sure you are up to date on the latest news in neurology. To give you a chance to catch up on any of the headlines you may have missed, we’ve compiled all the updates here. The coverage includes the latest FDA approvals, new designations, submissions, resubmissions, and clinical trial initiations and holds.

Clene Moves Toward Accelerated Approval NDA Filing for CNM-Au8 in ALS

At the beginning of the week, on May 5, 2026, Clene Nanoscience announced that it is preparing to submit a new drug application (NDA) under the FDA’s accelerated approval pathway for its investigational agent CNM-Au8 as a treatment for amyotrophic lateral sclerosis (ALS) following a recent Type C meeting with the agency.¹ The submission, expected in the third quarter of 2026, would be supported primarily by neurofilament light (NfL) biomarker data and prior phase 2 clinical studies.

The regulatory discussion is notable because the FDA indicated that the available dataset “may be capable” of supporting NDA review under accelerated approval and acknowledged that NfL could potentially serve as a reasonably likely surrogate endpoint to show benefit in ALS, a progressive neurodegenerative disease with limited disease-modifying treatment options.

“We are encouraged by the FDA’s careful evaluation of the benefits and risks associated with Clene’s ALS drug candidate, CNM-Au8, including the biomarker data the company provided,” Rob Etherington, MBA, President and CEO of Clene, in a statement.¹ “The filing of an NDA submission represents an important milestone for CNM-Au8 and for the ALS community. We are committed to working with the Agency on this filing and are conducting the Phase 3 confirmatory study for CNM-Au8, which we intend to commence in the first quarter of 2027.”

Efgartigimod Gains FDA Approval as First Treatment for Seronegative Forms of Myasthenia Gravis

A few days later, on May 8, 2026, 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. The approval extends the anti–FcRn therapy beyond its prior use in AChR-Ab–positive patients becoming the first approved treatment for the newly included seronegative population, marking a broader biomarker-independent use of the FcRn inhibitor in gMG.²

Efgartigimod’s approval stemmed largely from the results of phase 3 ADAPT SERON study (NCT06298552), a randomized, double-blind, placebo-controlled trial that featured 119 patients followed over a 5-week treatment period. The study is the largest study to date of patients with gMG who do not have detectable AChR-Ab across 3 serotypes. In the study, patients were randomly assigned to receive 4 once-weekly intravenous infusions of efgartigimod or placebo, with change in Myasthenia Gravis Activities of Daily Living as the primary end point, recorded at 29 days.

“Today’s approval means that all adult gMG patients, regardless of serotype, can now benefit from VYVGART’s rapid onset, sustained disease control, and favorable safety profile,” Luc Truyen, MD, PhD, chief medical officer at argenx, said in a statement.² "For clinicians, this simplifies treatment decisions, representing a major advancement in reaching as many patients living with gMG as possible.”

FDA Approves Ocrelizumab for Pediatric Patients With Relapsing-Remitting Multiple Sclerosis

On the same day, on May 8, 2026, 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 with often highly active disease.³

With the decision, ocrelizumab becomes an FDA-approved treatment option for pediatric RRMS, a group that accounts for an estimated 3% to 5% of MS cases globally and approximately 5000 to 10,000 children and adolescents in the United States. The approval was supported by data from the phase 3 OPERETTA 2 study (NCT05123703), which compared ocrelizumab with fingolimod (Gilenya; Novartis), previously the only FDA-approved therapy for pediatric RRMS.

“This approval represents a landmark for children living with MS in the US and their families, which can help close the longstanding gap in high-efficacy treatment options for children aged 10 and older,” Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development at Genentech, said in a statement.³ “By bringing a decade of efficacy and safety data to this younger population, Ocrevus may reduce relapses and potentially redefine what’s possible for their future.”

FDA Extends Review of Subcutaneous Starting Dose for Lecanemab in Early Alzheimer Disease

On the same day, on May 8, 2026, 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

According to the companies, the FDA requested additional information during its ongoing review and classified the submission as a “major amendment,” resulting in a standard 3-month extension to allow time for full evaluation of the updated materials. Importantly, Eisai and Biogen noted that the agency “has not raised any concerns to date regarding the approvability” of the subcutaneous starting-dose regimen.

If approved, the expanded indication would allow patients with mild cognitive impairment (MCI) or mild dementia due to AD to initiate treatment directly with a once-weekly subcutaneous autoinjector formulation rather than first receiving biweekly intravenous infusions. The proposed regimen builds on the FDA’s August 2025 approval of Leqembi Iqlik for maintenance dosing, which became the first at-home, self-administered antiamyloid treatment option for AD.

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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
2. 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
3. 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