Gene therapy trial for Duchenne muscular dystrophy put on hold by FDA

Written by Alexander Marshall

Gene therapy, SGT-001, to treat Duchenne muscular dystrophy halted by the FDA after second patient experiences serious adverse event.

The FDA has ordered Solid Biosciences (MA, USA) to halt their Phase 1/2 gene therapy clinical trial, IGNITE DMD, after a second patient has fallen ill in what has been classified as a serious adverse event. SGT-001, the novel adeno-associated viral vector being trialed, was hoped to combat Duchenne muscular dystrophy (DMD) but its future has been called into question following recent results.

Having been temporarily halted once previously, the FDA were quick to pause the trial again when a second patient presented with symptoms of complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury and cardio-pulmonary insufficiency.

The adeno-associated virus-based gene therapy was supposed to provide patients with a synthetic dystrophin gene, termed micro-dystrophin, to compensate for the loss of functionality in their own owing to the inherited mutation. Preclinical data obtained by Solid Biosciences did support the concept, demonstrating an ability to slow or stop the progression of DMD; however, the virus has now left one of the six patients enrolled on the trial under careful observation.

       “We are encouraged that this patient is recovering. I would like to thank both the patient and his family for their participation in our study, as well as the team at the University of Florida for the excellent care they provide,” stated Chief Executive Officer, Ilan Ganot (Solid Biosciences). “We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients. In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001. We look forward to sharing this additional data and working with the FDA to resolve the clinical hold and determining next steps for the program.”

DMD is genetic disease which results from the disruption of the dystrophin gene and results in gradual muscle decline. While the life expectancy has substantially increased in recent years, the condition still ends in respiratory failure leaving very few cases living beyond their 30s.

While the patient is improving, the damage may have already been done to Solid Biosciences, with their share price being substantially hit. Despite this, the company insist they are committed to reporting biomarkers data to the FDA and resolving the hold upon the study.


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