US FDA's Record-Breaking Month Of Breakthrough Therapy Designations

Seven weeks before its action date and a month after an advisory committee was hastily cancelled, Capricor gets a CRL for its cell therapy deramiocel in DMD-related cardiomyopathy citing efficacy and manufacturing concerns. It has a Phase III study nearing readout, though.

Bayesian statistics could help clinical trial sponsors to include external information in their analyses, but concerns around bias and incorrect conclusions remain, the European Medicines Agency said.

Industry associations EFPIA, EUCOPE and EURORDIS have praised the European Commission’s newly launched life sciences strategy, which will see investment funnelled into improving the clinical trial ecosystem and supporting advanced therapy development.

The Council of the EU’s vision for the pharma reform package includes a stronger role for member states regarding the supply of medicines and the prevention of indication-stacking for orphan drugs.