Solid Biosciences receives full clinical hold letter from FDA on IGNITE DMD
Solid Biosciences (SLDB) announced that the company has received a letter from the U.S. Food and Drug Administration relating to the previously-announced full clinical hold on IGNITE DMD, the company’s Phase I/II clinical trial for its investigational gene therapy, SGT-001, for the treatment of Duchenne muscular dystrophy.
The company plans to submit a response to this letter to the FDA in the coming weeks.
The FDA also informed Solid that the company has satisfactorily addressed manufacturing-related questions that led to the previously-announced partial clinical hold on the high dose of SGT-001 in IGNITE DMD.
“We believe SGT-001 has the potential to significantly benefit patients with DMD. We are working with the FDA to better understand the unexpected event that resulted in the clinical hold and put a plan in place to monitor and manage potential events in the future. I am happy to share that the treating physician has reported the patient is doing well,” said CEO Ilan Ganot.
“We are also pleased that we were able to lift the earlier partial clinical hold on the high dose of SGT-001 through our existing manufacturing process, which is an important ongoing development effort for our company.”
The full clinical hold was in response to the company’s report of a Serious Adverse Event in the first patient dosed with SGT-001 in IGNITE DMD, where several days after administration the patient was hospitalized due to laboratory findings that included a decrease in platelet count followed by a reduction in red blood cell count and evidence of complement activation.
The patient showed no signs or symptoms of coagulopathy and no relevant changes from baseline in liver function tests.
Following medical treatment, all of his laboratory parameters have either improved or returned to normal.
In its full clinical hold letter, the FDA requested additional information that is required prior to resuming the clinical trial, including an assessment of the underlying etiology of the event, the patient’s clinical status and laboratory parameters, and any additional measures to address patient safety. Solid will work closely with the FDA to address these questions.
SLDB closed at $10.56.
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