The FDA cleared Ultragenyx’s one-time AAV gene therapy Genglycos (pariglasgene brecaparvovec; DTX-401) for glycogen storage disease type Ia (GSDIa), giving the company its first marketable gene therapy. The accelerated approval covers patients 8 years and older and positions Ultragenyx for additional upside tied to its other late-stage rare-disease programs. In Europe, regulators ordered a recall of all batches of Amgen’s rare-disease drug Tavneos (avacopan) after the European Commission canceled its marketing authorization earlier this month. The action adds immediate commercial and clinical uncertainty for physicians and patients using the therapy. Separately, the FDA approved Regeneron’s Pasatru (garetosmab) for fibrodysplasia ossificans progressiva (FOP), opening a high-stakes commercial fight with Ipsen’s Sohonos. Regeneron’s label targets activin A to curb abnormal bone formation after a late-stage program reported large reductions versus placebo.