Gene Therapy
Arrowhead lipid-lowering drug latest data hits 'best-case scenario', severe hypertriglyceridemia indication expansion in sight
Arrowhead Pharmaceuticals announced that its gene medicine plozasiran met primary endpoints in two Phase III clinical trials for severe hypertriglyceridemia, with median reductions in triglyceride levels of 79% and 81% and a 78% reduction in acute pancreatitis events. The company plans to submit a supplemental application by the end of the year to expand the drug's indication. Analysts call this a 'best-case scenario'.
FDA Gene and Cell Therapy Review Chief Vijay Kumar to Step Down as Acting Director
Vijay Kumar, Acting Director of the Office of Gene and Cell Therapy Review at the FDA's Center for Biologics Evaluation and Research (CBER), will step down, with CBER Acting Director Karim Mikhail temporarily taking over the position to ensure regulatory continuity. Kumar will remain at the FDA. This change occurs during a period of high-level personnel instability at the FDA, following the resignation of Commissioner Marty Makary, the departure of CBER Director Vinay Prasad, and the dismissal of CDER Director Tracy Beth Høeg. Kumar's office reviewed experimental therapies from companies such as Uniqure, Regenxbio, and Atara Biotherapeutics, and his departure may impact the direction of rare disease drug review policies.