Duchenne Muscular Dystrophy Trial has Gotten the Go Ahead from the FDA to Continue Dosing

Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called IGNITE and is investigating SGT-001 as a therapy for the condition.

The hold was first placed in July of 2020 after the FDA requested updated safety data, efficacy data, manufacturing information, and direction on the amount that should be administered to each participant.

Solid has adequately responded to all of the FDA’s questions, and the trial has now been resumed. Researchers expect that this investigation should finish all dosing by the 1st quarter of next year.


Solid has reduced the number of empty viral capsules which allows fewer particles to be used while still achieving the target dose. Additionally, this improves the safety of the therapy. Solid sent the FDA data from their in vitro assay to demonstrate how this new approach varies from their original approach.

Solid also has reduced the maximum weight limit of their next 2 participants to 18kg. The outcomes from the patients will drive new direction for the study. If all goes well, the maximum weight could be increased for future participants as well.

Additionally, submitted to the FDA were safety data and efficacy data reports for all patients in the trial.

No additional AEs have been found in the 30 months after dosing. However, to continue to mitigate risk of AEs, IGNITE is adding prophylactic use of eculizumab and C1 esterase, as well as increasing the dose of prednisone in the first month following dosing.


This therapy is an AAV vector-mediated gene therapy that aims to cure the underlying cause of DMD. The therapy has already received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation by the FDA.

SGT-001 delivers microdystrophin, a synthetic dystrophin gene to the body. Preclinical data has shown that this therapy has potential to not only slow progression of disease, but stop it all together. Additionally, this therapy has been shown to work regardless of the genetic mutation present or the disease stage of the patient.

You can read more about this study here.

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