Why Solid Biosciences Is Under Fire From the FDA

Health and Healthcare

Published: March 15, 2018 10:25 am

Last Updated: January 12, 2020 2:24 am

Solid Biosciences Inc. (NASDAQ: SLDB) watched its shares get crushed on Thursday after the company announced some complications with the U.S. Food and Drug Administration (FDA). Specifically, the FDA notified the firm that its Phase 1/2 clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD) has been placed on clinical hold.

The study was designed to assess the safety and efficacy of SGT-001 in ambulatory and non-ambulatory children and adolescents with DMD. The first patient dosed in the clinical trial was a non-ambulatory adolescent who received SGT-001 on February 14, 2018. 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.

At that time, the patient showed no signs or symptoms of coagulopathy (bleeding disorder) and no relevant changes from baseline in liver function tests. The patient responded well to medical treatment and is currently asymptomatic. All laboratory parameters have either improved or returned to normal, and he is continuing outpatient assessments per protocol.

Solid Biosciences reported the event to the FDA and, because it was unexpected, classified it as a Suspected Unexpected Serious Adverse Reaction (SUSAR). The FDA informed the company that the clinical hold was due to the event.

The company has halted enrollment and dosing in the Ignite DMD study and is awaiting the formal Clinical Hold letter from the FDA to understand the requirements for resuming the clinical trial.

Shares of Solid Biosciences were last seen down more than 63% at $9.68, with a consensus analyst price target of $37.40 and a new 52-week range of $9.52 to $33.74.

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