Sarepta to launch late-stage study of high-dose eteplirsen

Sarepta Therapeutics (SRPT -0.9%) is poised to start a Phase 3 clinical trialevaluating eteplirsen in ~144 DMD patients. The trial should commence this month and wind up in October 2024. It will assess higher doses of the antisense oligonucleotide than the current approved dose of 30 mg/kg of body weight, however.

The FDA initially approved the drug, branded as EXONDYS 51, almost three years ago under accelerated review status with the requirement that the company conduct a pivotal Phase 3 study to prove efficacy and secure full approval. Failure to demonstrate a statistically valid treatment benefit would potentially be the basis for withdrawing the product from the U.S. market.

https://seekingalpha.com/news/3492298-sarepta-launch-late-stage-study-high-dose-eteplirsen