The FDA cleared Regeneron’s gene therapy Otarmeni (lunsotogene parvec) for a rare inherited form of hearing loss caused by otoferlin variants, marking the first FDA approval for a gene therapy under the agency’s National Priority Voucher program. Regeneron said it will offer the therapy at no cost to eligible patients in the U.S., with the approval reflecting the program’s push to accelerate novel modalities into clinical care. In a separate regulatory update, Novartis withdrew an EU filing seeking to expand Pluvicto’s prostate cancer indication into earlier lines of treatment, even though the same expansion is already approved in the U.S. and the U.K. The decision underscores how label expansion trajectories can diverge across regulators despite comparable evidence bases. Meanwhile, Sanofi’s tolebrutinib is positioned for European approval after EU regulators recommended it for a subset of MS patients following an FDA complete response letter. The split between U.S. and European outcomes points to ongoing differences in benefit–risk framing and safety interpretation for BTK inhibitor programs in neurology.