AAVantgarde gets orphan drug status for Stargardt gene therapy

The U.S. FDA has granted AAVantgarde Bio orphan drug designation for AAVB-039, the company’s investigational gene therapy for the treatment of Stargardt disease secondary to biallelic mutation in ABCA4.

The company has also received clinical trial authorization (CTA) approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA).

The most common form of inherited juvenile macular degeneration, Stargardt is caused by mutations in the ABCA4 gene. The condition leads to the progressive loss of central vision.

AAVantgarde’s AAVB-039, which was granted FDA fast track designation back in August, addresses the underlying genetic cause of the disease by providing the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations. The AAV gene therapy is currently being evaluated in the phase 1/2 CELESTE clinical trial, which is assessing safety, tolerability, and preliminary efficacy in patients with Stargardt disease across three dose levels.