Yesterday, Sarepta Therapeutics announced that the FDA has issued a Clinical Hold letter for the company’s phase I/II trial testing the safety and effectiveness of delivering a small but functional dystrophin gene (micro-dystrophin) to the muscles using a harmless virus.

Clinical holds effectively pause a clinical trial and can be used for a variety of reasons, including unexpected events, or serious side effects. In this case, the clinical hold is due to an “out-of-specifciation” lot of gene therapy material. Batches of medicine and trial treatments are made to exacting specifications that are monitored carefully to ensure the safety of the people who receive them. If the specifications of a lot are not perfect, then a clinical hold can be issued to allow for investigation of the problem.

Sarepta has already identified the cause of the issue and with the Research Institute at the Nationwide Children’s Hospital (where the trial is taking place) has come up with a plan to address it. If the FDA approves the plan, Sarepta plans to continue the trial as soon as possible and still hopes to start a larger trial late this year and we’ll keep you updated as we hear more.

In a letter to the community, Doug Ingram, Sarepta’s president and CEO said:

The clinical hold resulted from an out-of-specification lot of gene therapy material… we understand the source of the issue, have been given clear guidance on how to address it, and have a plan to keep our clinical program on track.

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