Sarepta resumes dosing at U.K. sites after DMD trial halt

Sarepta Therapeutics Inc. (NASDAQ:SRPT) President and CEO Doug Ingram said the company has resumed dosing at U.K. sites in the Phase III ESSENCE trial evaluating golodirsen (SRP-4053) and casimersen (SRP-4045) to treat Duchenne muscular dystrophy amenable to exon 53 or 45 skipping, respectively. During a presentation Tuesday at the Barclays Global Healthcare Conference in Miami, Ingram said the U.K.'s Medicine and Healthcare products Regulatory Agency (MHRA) reviewed the study and allowed dosing to resume.

Sarepta had halted dosing at U.K. study sites last month as a result of a serious adverse event (SAE) that triggered an MHRA stopping rule, Ingram said. The SAE was rhabdomyolysis and elevated creatine kinase levels, both of which are common in DMD patients, he noted (see BioCentury Extra, Feb. 9)...