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In May, the FDA issued several approvals, accepted new drug applications (NDAs) for review, and granted expedited regulatory designations for therapies to treat a range of neurologic conditions, including autoimmune disorders, neurodegenerative diseases, stroke, and rare genetic syndromes. For information on approvals in other specialties during the first quarter of 2026, see the Medscape Medical News FDA Drug Approvals page.

Efgartigimod Use for Seronegative Generalized Myasthenia Gravis (gMG) Expanded

On May 8, the FDA expanded the indication for efgartigimod alfa (Vyvgart; argenx) and hyaluronidase-qvfc (Vyvgart Hytrulo) to include adults with anti-acetylcholine receptor (anti-AChR) antibody-negative gMG. As reported by Medscape Medical News, the therapy is the first and only neonatal Fc receptor blocker approved for both antibody-positive and antibody-negative gMG. The approval was based on data covered previously by Medscape Medical News from the phase 3 ADAPT SERON trial — the largest study to date in patients with AChR antibody-seronegative gMG.

First B-Cell-Depleting Therapy Approved for Pediatric Relapsing-Remitting Multiple Sclerosis (RRMS)

Also on May 8, the FDA approved intravenous ocrelizumab (Ocrevus; Genentech) for pediatric patients aged 10 years or older with RRMS. It is the first anti-CD20 therapy approved in the US for this patient population, Medscape Medical News reported. The decision was supported by findings from the phase 3 OPERETTA 2 trial comparing ocrelizumab with fingolimod, which was previously the only FDA-approved therapy for pediatric RRMS.

Soluble TNF (sTNF) Inhibitor for Early Alzheimer’s disease (AD) Moves Forward

FDA awarded Fast Track designation to a first-in-class treatment for early AD on May 14. XPro1595 (pegipanermin; INmune Bio Inc.) is a selective sTNF inhibitor designed to reduce neuroinflammation while preserving normal immune signaling in patients with AD-related mild cognitive impairment or AD dementia. In a statement, the company said the next step in development is a phase 2b/3 registrational study in patients with early AD and biomarkers of inflammation.

Prader-Willi Syndrome Program on Clinical Hold

On May 14, the FDA placed a hold on clinical trials of ARD-101 (Aardvark Therapeutics), an investigational therapy for hyperphagia in Prader-Willi syndrome, after reported cardiac safety concerns observed in a healthy volunteer study. The action follows the company voluntarily pausing its phase 3 HERO trial and a related open-label extension study, the manufacturer reported in a statement, adding that they are working with the FDA to determine next steps for development of the drug.

Factor XIa Inhibitor Advances in Secondary Stroke Prevention

The FDA accepted an NDA and granted priority review for asundexian, an oral factor XIa inhibitor, for the prevention of recurrent ischemic stroke in patients following a non-cardioembolic ischemic stroke or transient ischemic attack, manufacturer Bayer reported in a statement on May 19. The application is supported by results from the phase 3 OCEANIC-STROKE trial, which showed that treatment with asundexian was associated with a significant reduction in recurrent ischemic stroke when added to standard antiplatelet therapy, as reported by Medscape Medical News.

Modified Amino Acid for Ataxia-Telangiectasia (A-T) Therapy Gets Priority Review

A supplemental NDA for levacetylleucine (AQNEURSA; IntraBio) for the treatment of A-T in adult and pediatric patients received FDA acceptance and priority review on May 19. IntraBio noted that the FDA’s priority review designation reflects the urgency of an unmet need for this patient population. A PDUFA target date of September 19 was assigned to the application. The application is supported by data from the phase 3 ATTeST trial, which showed significant improvements in neurologic symptoms and functional outcomes in patients aged 4 years or older with confirmed A-T, Medscape Medical News reported.