The Food and Drug Administration (FDA) has granted Orphan Drug designation to SBT101 for the treatment of patients with adrenomyeloneuropathy (AMN), a progressive and debilitating spinal cord disease caused by mutations in the ABCD1 gene.

SBT101 is an adeno-associated virus (AAV) gene therapy candidate designed to compensate for the ABCD1 mutation, to increase ABCD1 expression, and reduce very long chain fatty acid levels. In preclinical studies, SBT101 was associated with a dose-dependent improvement of AMN disease markers in mouse models and was found to be well tolerated in nonhuman primates at 6 months post-treatment.

The Company will initiate a randomized, controlled phase 1/2 trial to evaluate the efficacy and safety of SBT101 in patients with AMN in the second half of 2022. The FDA previously granted Fast Track designation to SBT101 for this indication. 

“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.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.

Reference

FDA grants Orphan Drug Designation to SBT101, the first investigational AAV-Based gene therapy for adrenomyeloneuropathy (AMN). News release. SwanBio Therapeutics. Accessed March 15, 2022. https://www.businesswire.com/news/home/20220315005309/en/FDA-Grants-Orphan-Drug-Designation-to-SBT101-the-First-Investigational-AAV-Based-Gene-Therapy-for-Adrenomyeloneuropathy-AMN