FDA Grants Fast Track Designation to SwanBio for AMN Gene Therapy

The U.S. Food and Drug Administration granted Fast Track designation to SwanBio Therapeutics for its lead experimental gene therapy SBT101 for the treatment of adrenomyeloneuropathy.

Photo: Tom Anderson, CEO and director of SwanBio Therapeutics

Adrenomyeloneuropathy (AMN) is the adult-onset degenerative spinal cord disease that affects people living with adrenoleukodystrophy (ALD), a category of rare, genetic, and metabolic conditions. AMN is characterized by progressive loss of mobility, incontinence, and debilitating pain. It affects adults with mutations in the ABCD1 gene, which encodes a protein essential to the processing and breakdown of very long chain fatty acids (VLCFA). Without a functioning version of this protein there is an accumulation of VLCFA to toxic levels that leads to progressive dysfunction of the central nervous system. Between 8,000-10,000 men in the U.S. and E.U. are living with AMN.

“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, CEO 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.”

SBT101 is the first AAV-based gene therapy in development designed to compensate for the disease-causing ABCD1 mutation, to increase ABCD1 expression, and reduce very long chain fatty acid (VLCFA) levels specifically for people living with adrenomyeloneuropathy. In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of AMN disease markers in mouse models and was shown to be well-tolerated in non-human primates at six months post-treatment.

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.

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.

Author: Rare Daily Staff