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Solid Biosciences' SGT-003 Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy

• Solid Biosciences' SGT-003, a next-generation gene therapy, has been granted Fast Track designation by the FDA for treating Duchenne muscular dystrophy (DMD). • The Fast Track designation aims to accelerate the development and review of SGT-003, potentially offering a substantial improvement over existing DMD therapies. • SGT-003 leverages Solid Biosciences' proprietary AAV-SLB101 capsid, showing increased biodistribution to cardiac and skeletal muscle in preclinical studies. • A Phase I/II clinical study is planned to assess the safety and tolerability of SGT-003 in pediatric DMD patients, with long-term efficacy evaluated over five years.

Solid Biosciences Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SGT-003, its next-generation gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD). This regulatory milestone is designed to expedite the development and review process for drugs that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.
SGT-003 is being developed utilizing Solid Biosciences’ proprietary and rationally designed capsid, AAV-SLB101. Preclinical data has demonstrated that SGT-003 exhibits increased biodistribution to cardiac and skeletal muscle, including the diaphragm, compared with AAV9 in nonhuman primates. This enhanced distribution could potentially lead to improved therapeutic outcomes for DMD patients.
The Fast Track designation facilitates closer communication between the FDA and Solid Biosciences, aiming to improve the efficiency of product development and accelerate the delivery of this novel therapeutic to patients. Solid Biosciences' stock experienced a significant rally following the announcement, reflecting investor optimism regarding the regulatory update for SGT-003.

Clinical Development Plans

In November 2023, the FDA cleared Solid Biosciences’ Investigational New Drug (IND) application, allowing the company to initiate clinical studies on SGT-003 for DMD. The planned Phase I/II SGT-003-101 study will evaluate the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. The therapy will be administered as a one-time intravenous injection across two cohorts, each with a minimum of three patients. The study design also includes a provision for potential cohort expansion if deemed necessary.
Cohort 1 of the study will focus on patients aged 4 to under 6 years with DMD. The long-term safety and efficacy of SGT-003 will be evaluated for a total of five years post-treatment. DMD is a progressive and degenerative disorder characterized by muscle weakness and wasting.

Current Treatment Landscape

Currently, Sarepta Therapeutics markets the only approved one-shot gene therapy drug in the United States for DMD. The FDA granted accelerated approval to Sarepta's Elevidys (delandistrogene moxeparvovec or SRP-9001), an adeno-associated virus-based gene therapy, to treat ambulatory pediatric patients aged between four and five years with DMD in June 2023. SGT-003 represents another promising avenue for addressing this debilitating condition.
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Reference News

[1]
Solid Biosciences' (SLDB) DMD Drug Gets Fast Track Tag, Stock Up
finance.yahoo.com · Dec 8, 2023

Solid Biosciences Inc. received FDA Fast Track designation for SGT-003, a next-generation gene therapy for Duchenne musc...

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