Solid Biosciences has announced that the first patient to be treated in its phase 1/2 clinical trial to test SGT-001, the company’s experimental gene therapy treatment for DMD, experienced a serious safety issue that was not among what was predicted based on what is known about the therapy — known as a Suspected Unexpected Serious Adverse Reaction.
The incident occurred several weeks ago and the patient appears to be healthy at this point. The U.S. Food and Drug Administration (FDA) has put the clinical trial on hold and will work with Solid Biosciences to understand what happened and to determine whether it is safe to continue with the study. MDA is in contact with Solid Biosciences and will share more as information becomes available.
Letter to the community from Solid Biosciences:
Dear Duchenne community members,
Today we announced that the U.S. Food and Drug Administration has placed our Phase I/II clinical trial for SGT-001, IGNITE DMD, on Clinical Hold following a serious adverse event that occurred in the first patient dosed, a non-ambulatory adolescent. The patient was admitted to the hospital, received treatment and, as of the writing of this letter, is home with his family with no symptoms. Details about the event can be found in the press release we issued today, which is available here. The team at Solid will be working with the principal investigator and FDA to fully understand the cause and nature of this event, as well as identify appropriate next steps as soon as possible.
Since our inception, we have been focused on working to bring meaningful treatments to patients with Duchenne muscular dystrophy (DMD). This cause is deeply personal to our company, which was founded and is led by individuals touched by DMD, and we are committed to identifying and bringing medicines to patients safely and responsibly. These principles underlie everything we do as an organization, and we take them seriously.
We greatly appreciate the trust placed in us by patients and families and will honor that through our dedication and work to end this disease. We appreciate your patience while we work to resolve this situation.