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FDA Imposes Clinical Hold on Solid Biosciences' DMD Gene Therapy Following Adverse Event

The FDA has placed a clinical hold on Solid Biosciences' IGNITE DMD trial for its Duchenne muscular dystrophy (DMD) gene therapy, SGT-001, after a serious adverse event was reported in a participant. This marks the second clinical hold for the trial, raising concerns over the therapy's safety profile. The event involved complement activation and other severe reactions, prompting a significant drop in Solid Bio's share price.

FDA Halts Solid Biosciences' DMD Gene Therapy Trial

The U.S. Food and Drug Administration (FDA) has issued a clinical hold on Solid Biosciences' IGNITE DMD trial, a study investigating the gene therapy SGT-001 for Duchenne muscular dystrophy (DMD). This decision follows a serious adverse event in a seven-year-old participant, the sixth child to receive the therapy and the third to be treated with the highest dose (2x1014vg/kg) planned for the study.

Details of the Adverse Event

The adverse event was characterized by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury, and cardio-pulmonary insufficiency. Solid Biosciences' CEO, Ilan Ganot, reported that the child is responding to treatment. This incident echoes a previous clinical hold placed on the trial last year after a similar adverse event occurred in the first patient treated with a lower dose of SGT-001.

Implications for the Trial

This latest hold raises concerns about the safety profile of SGT-001, especially at higher doses. The trial's protocol has been amended to allow the use of Alexion’s complement inhibitor Soliris (eculizumab) if needed, following its successful use in resolving toxicity in the previous case. However, the recurrence of serious adverse events has led to a significant drop in Solid Bio's share price, reflecting investor anxiety over the future of the DMD gene therapy program.

Looking Ahead

Solid Biosciences plans to collaborate closely with the FDA to address the clinical hold and determine the next steps for the IGNITE DMD trial. Data from two additional patients who received the higher dose of SGT-001 are expected by the end of the year, which may provide further insights into the therapy's safety and efficacy.
SGT-001 aims to deliver a functional version of dystrophin, a protein crucial for muscle function, using an adeno-associated virus (AAV) vector. The therapy represents a promising approach to treating DMD, a genetic disorder characterized by progressive muscle degeneration and weakness.
This development occurs amidst a competitive landscape for DMD gene therapies, with companies like Sarepta and Pfizer also advancing their candidates. The outcome of Solid Biosciences' efforts to resolve the clinical hold and proceed with the IGNITE DMD trial will be closely watched by the medical and investment communities alike.
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Reference News

[1]
Solid Bio hit as FDA slaps clinical hold on DMD gene therapy
pharmaphorum.com · Nov 12, 2019

The FDA has placed a clinical hold on Solid Biosciences' Duchenne muscular dystrophy gene therapy, SGT-001, after a seri...

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