FDA Grants Orphan Drug Designation to SBT101, the First Investigational AAV-Based Gene Therapy for Adrenomyeloneuropathy (AMN)

PHILADELPHIA--(BUSINESS WIRE)-- SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the company’s lead candidate, SBT101. SBT101 is being investigated as a potential treatment for patients with adrenomyeloneuropathy (AMN). SwanBio plans to initiate a randomized, controlled Phase 1/2 clinical trial to assess the safety and efficacy of SBT101 in the second half of 2022.

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. There are no approved treatment options available today for people with AMN.

“The FDA’s decision to grant Orphan Drug Designation to SBT101 underscores the urgent unmet need for novel therapies for people living with this devastating condition that affects adults in the prime of their lives,” said Tom Anderson, chief executive officer and director of SwanBio Therapeutics. “This designation, paired with the FDA’s recent clearance of our Investigational New Drug application and decision to grant Fast Track designation for SBT101, builds strong momentum for SwanBio as we advance toward clinical initiation later this year.”

ODD is granted by the FDA based on the review of preclinical data from investigational treatments for rare diseases, which are defined as conditions affecting fewer than 200,000 people in the United States. This designation qualifies the sponsor of the drug for certain incentives, including most notably, seven years of marketing exclusivity upon drug approval from the FDA.

About SBT101
SBT101 is the first AAV-based gene therapy candidate cleared by the FDA for human studies, designed to compensate for the disease-causing ABCD1 mutation in people living with adrenomyeloneuropathy (AMN). 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. The clinical program for SBT101 builds on this positive preclinical data, plus the company’s deep understanding of the underlying pathophysiology of the disease and the patient experience of AMN, including new insights being gathered in an ongoing natural history study.

SwanBio expects to initiate a randomized, 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. Earlier this year, the FDA cleared SwanBio’s Investigational New Drug application for SBT101 and granted SBT101 Fast Track Designation.

About Adrenomyeloneuropathy
Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction which all affect quality of life. Between 8,000-10,000 men in the United States and European Union are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom control.

About SwanBio Therapeutics
SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications – spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBio’s lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220315005309/en/