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CHARLESTOWN, Mass. - Solid Biosciences Inc. (NASDAQ:SLDB), a biotechnology company with a market capitalization of $483 million, announced Wednesday it has received Fast Track designation from the U.S. Food and Drug Administration for its gene therapy candidate SGT-501, designed to treat catecholaminergic polymorphic ventricular tachycardia (CPVT). According to InvestingPro data, the company’s stock has shown strong momentum with an 11.7% gain over the past week.
The designation adds to SGT-501’s previously received Orphan Drug and Rare Pediatric Disease designations. Fast Track status facilitates more frequent interactions with the FDA and potential eligibility for priority review.
CPVT is a life-threatening cardiac condition affecting approximately 1 in 10,000 people globally. The condition causes abnormal heart rhythms triggered by physical activity or emotional stress, which can lead to fainting, seizures, and sudden death.
SGT-501 is designed to deliver a functional copy of the human cardiac calsequestrin gene to heart muscle cells, addressing the underlying calcium dysregulation seen in CPVT. The therapy received FDA Investigational New Drug clearance and Health Canada Clinical Trial Application approval on July 8.
"FDA IND clearance and Fast Track designation provide important scientific validation of SGT-501 and recognition of the continuing and severe unmet needs posed by CPVT," said Jessie Hanrahan, Chief Regulatory & Preclinical Operations Officer at Solid Biosciences.
The company plans to initiate a Phase 1b clinical trial in the fourth quarter of 2025. The study will be a first-in-human, open-label, multicenter trial evaluating the safety, tolerability, and efficacy of SGT-501.
Currently, there are no FDA-approved treatments that address the underlying mechanisms of CPVT.
The information in this article is based on a press release from Solid Biosciences.
In other recent news, Solid Biosciences has received FDA approval for its Investigational New Drug (IND) application for SGT-501, a gene therapy targeting catecholaminergic polymorphic ventricular tachycardia (CPVT). Health Canada has also approved the company’s clinical trial application for this therapy. This development comes amidst safety concerns with Sarepta Therapeutics’ Duchenne muscular dystrophy treatment, Elevidys, which led to an FDA investigation and a black box warning. In light of these concerns, Citi has maintained its Buy rating on Solid Biosciences, setting a price target of $14.00. Cantor Fitzgerald has reiterated an Overweight rating with a $16.00 price target after meetings with Solid Biosciences’ management. JMP Securities also continues to rate the stock as Market Outperform, with a price target of $15.00, citing the larger AAV-naive Duchenne muscular dystrophy patient population as a positive factor. These ratings reflect continued confidence in Solid Biosciences despite the competitive landscape challenges.
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