RARE Daily

FDA Grants Fast Track Designation to Solid Biosciences for Duchenne Muscular Dystrophy Gene Therapy

December 7, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Solid Biosciences for SGT-003, the company’s next-generation Duchenne muscular dystrophy gene therapy candidate.

The Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

“Receipt of FDA Fast Track designation underscores the importance of rapidly developing SGT-003 to potentially aid the unmet needs of the Duchenne community,” said Bo Cumbo, president and CEO at Solid Biosciences. “Having received IND clearance for SGT-003 last month, we are pleased to be expediting the development of a potentially life-changing therapy and look forward to continuing to work closely with the FDA.”

Duchenne muscular dystrophy (DMD) is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone.

SGT-003 uses a proprietary, rationally designed capsid to deliver a DNA sequence encoding a shortened form of the dystrophin protein containing the R16-R17 nNOS binding domain. Preclinical data suggests this may be important for both muscular function and durability of benefit in patients.

The FDA cleared Solid’s Investigational New Drug Application in November 2023. The planned phase 1/2 trial, SGT-003-101, is a first-in-human, open-label, multicenter trial to determine the safety and tolerability of SGT-003 in pediatric patients with DMD at a dose of 1E14vg/kg. SGT-003 will be administered as a one-time intravenous infusion to patients in two cohorts with a minimum of three patients each, with the potential for cohort expansion. Cohort 1 will study patients aged 4 to more  6 years of age with DMD. Long-term safety and efficacy will be evaluated for a total of 5 years following treatment.

“We appreciate the FDA’s partnership and their recognition of the continuing unmet need in the DMD community,” said Jessie Hanrahan, chief regulatory officer at Solid Biosciences. “We look forward to frequent engagements with the Agency to discuss development of SGT-003 and ensure that our clinical development program will appropriately collect the data needed to support review of SGT-003 for future marketing authorization.”

Photo: Jessie Hanrahan, chief regulatory officer at Solid Biosciences

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