Adrenomyeloneuropathy Gene Therapy Gets Orphan Drug Designation


The new designation follows an IND clearance and fast track review in February 2022.

The FDA has granted orphan drug designation to SwanBio Therapeutics’ investigational adeno-associated virus (AAV) vector-based gene therapy SBT101 for the potential treatment of adrenomyeloneuropathy (AMN).1

“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,” Tom Anderson, chief executive officer and director, SwanBio Therapeutics, said in a statement.1

SBT101 is the first investigational AAV gene therapy for AMN. It increases ABCD1 expression levels and reduces very long chain fatty acid (VLCFA) levels in AMN by delivering a corrected ABCD1 gene. This approach has shown efficacy in previous preclinical studies. SwanBio plans to assess the safety and efficacy of SBT101 in humans in a randomized, controlled phase 1/2 clinical trial in the second half of 2022.

“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,” Anderson said in an earlier statement.2

READ MORE: FDA Recognizes Canavan Disease Gene Therapy With Multiple Designations

SwanBio has started 2022 off strong, with SBT101’s investigational new drug application receiving clearance in early February 2022, quickly followed by the FDA granting the therapy a fast-track review in late February.2,3

“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,” Anderson added.1

To better understand AMN and the natural progression of the disease,SwanBio is conducting the natural history CYGNET study (NCT05008874) in adult men with AMN. The study hopes to elucidate quality-of-life, and AMN-related disabilities that may arise in the disease course. Findings from the study may also inform future research and clinical development of therapies targeting AMN. CYGNET continues to recruit, with a goal of 80 participants.

"SBT101 has the potential to become the first disease-modifying treatment for patients with AMN, a devastating and progressive disease with no approved treatments. We look forward to initiating clinical development of SBT101 later this year, bringing us closer to our ultimate goal of delivering life-changing treatments to patients,” Anderson previously said.3

1. FDA grants orphan drug designation to SBT101, the first investigational AAV-based gene therapy for adrenomyeloneuropathy (AMN). News release. SwanBio Therapeutics. March 15, 2022.
2. FDA grants fast track designation to SBT101, the first investigational AAV-based gene therapy for patients with adrenomyeloneuropathy (AMN). News release. SwanBio. February 16, 2022.
3. SwanBio Therapeutics Announces FDA investigational new drug clearance for first AAV-based gene therapy for the treatment of adrenomyeloneuropathy. News release. SwanBio Therapeutics. January 25, 2022.
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