Pharmaceutical Business review

SwanBio gets FDA Fast Track designation for adrenomyeloneuropathy therapy

AMN is an adult-onset degenerative spinal cord disease which affects adrenoleukodystrophy patients.. Credit: Frank Gaillard / commons.wikimedia.org.

SBT101 is in development for compensating the disease-causing ABCD1 mutation.

It is designed for reducing very long chain fatty acid (VLCFA) levels and increase ABCD1 expression for AMN people.

AMN is an adult-onset degenerative spinal cord disease which affects adrenoleukodystrophy (ALD) patients.

It is caused by mutations in the ABCD1 gene that lead to mobility loss, incontinence, and pain.

The company is planning to commence a placebo-controlled, randomised Phase I/II clinical study of the therapy to assess its efficacy and safety in AMN patients.

The clinical trial is set to start in the second half of this year.

SwanBio stated that SBT101’s clinical programme builds on its understanding of AMN, along with the new insights gathered in CYGNET, the ongoing natural history study of the company.

SwanBio Therapeutics CEO and director Tom Anderson said: “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.

“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.”