The FDA Puts a Halt to Trial for SGT-001, A Treatment in Development for Duchenne Muscular Dystrophy

According to a story from, the FDA had to order the cessation of Phase II/III trials for the microdystrophin transfer therapy SGT-001, which was in development for the treatment of Duchenne muscular dystrophy. The stop order was announced by Solid Biosciences, Inc., the company responsible for its development.
Duchenne muscular dystrophy is the most severe and commonly occurring for of muscular dystrophy, a genetically linked disorder in which the muscles of the upper legs, upper arms, and pelvis begin to atrophy. Most people with the condition are unable to walk by age twelve; the condition shortens average life expectancy to about 26 years old, with fatal complications mostly involving the heart and breathing muscles. You can learn more about Duchenne muscular dystrophy here.
The clinical trial for SGT-001 was brought to a hold not long after it began; the first patient receive a dose with the treatment had to be sent to the hospital after lab examinations identified an reduction in platelet count and then a further decrease in red blood cell count. After the company reported the occurrence to the FDA, the agency immediately categorized the incident as a Suspected Unexpected Serious Adverse Reaction. This classification can be used for any such event that presents potentially severe side effects that were not in any way anticipated before the trial began.

The hold order comes as bad news for Solid Biosciences, as SGT-001 was the company’s lead drug candidate. In preclinical assessments, data suggested that that treatment could potentially slow down or even stop disease progression; SGT-001 is meant to supplement the lack of dystrophin in the patient’s muscles, which is a major cause of the muscle loss that occurs in Duchenne muscular dystrophy.

The only slight consolation is that the patient in question responded well to the treatment and currently is not experiencing symptoms. Nevertheless, all dosing and enrollment operations for the clinical trial are being discontinued indefinitely. In these situations, it is the responsibility of the company to figure out what happened, and Solid Biosciences will be forced to wait for an official letter sent from the FDA in order to understand the necessary requirements for the trial to restart.

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