The week in pharma: action, reaction and insight - week to November 26

By Barbara Obstoj-Cardwell

Last week’s notable news included US biotech BioMarin Pharmaceuticals receiving Food and Drug Administration approval for its Voxzogo in the treatment of achondroplasia, the most common form of dwarfism. Arrowhead Pharmaceuticals signed a heavily back-loaded agreement with the UK’s GlaxoSmithKline for its non-alcoholic steatohepatitis (NASH) candidate ARO-HSD, marking the British firm’s first foray into the sector that has seen many disappointments. Among regulatory developments, Kura Oncology had a setback last Tuesday, when the FDA put a partial clinical hold on its acute myeloid leukemia (AML) trial of KO-539 following a patient death. Finally, Ionis Pharma and partner Pfizer released Phase IIb clinical trial results for their cholesterol lowerer vupanorsen that, while meeting its primary endpoint, raised a number of questions.

BioMarin: Voxzogo launch to benefit from company experience