The week in pharma: action, reaction and insight – week to August 16, 2024

By Barbara Obstoj-Cardwell. Editor

Last week’s research news included US biotech Avidity Biosciences releasing strong early-stage trial results for its Duchenne muscular dystrophy (DMD) candidate delpacibart zotadirsen (AOC 1044). On the regulatory front, the US Food and Drug Administration (FDA) approved Ascendis Pharma’s hypoparathyroidism drug Yorvipath. The FDA also granted approval for Gilead Sciences’ seladelpar, now trade-named Livdelzi, for the treatment of the rare liver disease PCB. Also of significant importance, the US Centers for Medicare and Medicaid Services (CMS) last week selected 10 drugs for the first cycle of negotiations for pricing under the Inflation Reduction Act (IRA).

Del-Zota delivers in DMD, Avidity to engage with regulators