FDA Grants Fast Track Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Patients With Adrenomyeloneuropathy (AMN)

SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN).

AMN is an adult-onset progressive, degenerative spinal cord disease caused by mutations in the ABCD1 gene leading to loss of mobility, incontinence and pain. SwanBio plans to initiate a randomized, placebo-controlled Phase 1/2 clinical trial designed to assess the safety and efficacy of SBT101 in patients with AMN in the second half of 2022. The clinical program for SBT101 builds on SwanBio’s unique understanding of AMN, including new insights being gathered in the company’s ongoing natural history study, CYGNET.

“People living with AMN currently rely on a combination of symptom control, physical therapy and mobility aids, with no approved treatment to slow or alter the progression of this debilitating disease,” said Tom Anderson, chief executive officer and director of SwanBio Therapeutics. “The FDA’s decision to grant Fast Track designation for SBT101, following the recent clearance of our investigational new drug application for the program, further underscores the serious and unmet need for an effective treatment for AMN. We look forward to continuing to advance SBT101 as we work toward our goal of bringing life-changing treatments to patients.”

The Fast Track process is designed to facilitate the development and expedite the review of investigational treatments that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions. Programs with Fast Track designation can benefit from early and frequent communication with the FDA throughout the entire drug development and review process, helping to ensure the collection of appropriate data needed to support a drug approval application.

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