Sarepta Therapeutics’ long-awaited confirmatory study of two of its ‘exon-skipping’ therapies for forms of Duchenne muscular dystrophy failed to meet its primary endpoint, the company

One day after stepping down as CDER Director, George Tidmarsh has said he will fight the investigations into his conduct and is reconsidering his decision.

Two federal district courts have reached opposite conclusions in cases challenging states’ authority to regulate how drugmakers distribute 340B discounts. An Oklahoma federal judge on

Eli Lilly plans to build another facility to make its oral GLP-1 drug orforglipron. The $3 billion site will be located in Katwijk, the Netherlands.

The UK’s medicines regulatory agency said it is working to “overhaul” its framework for approving rare disease therapies. The Medicines and Healthcare products Regulatory Agency