US-based biopharmaceutical company PTC Therapeutics Inc (NASDAQ: PTCT) announced on Wednesday that it has received accelerated approval from the US Food and Drug Administration (FDA) for its gene therapy for the treatment of AADC deficiency, a highly morbid and life-shortening rare genetic disorder that results in the inability to synthesise dopamine, a neurotransmitter essential for motor function.

According to PTC Therapeutics this is the first-ever gene therapy approved in the United States that is directly administered to the brain.

The gene therapy will be marketed in the United States with the brand name KEBILIDI (eladocagene exuparvovec-tneq) and is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity. The company is in the process of launching the therapy, with centres of excellence already identified and surgeons trained in the procedure to deliver it.

KEBILIDI received accelerated approval based on the safety and clinical efficacy findings in the ongoing global clinical trial of the therapy. Confirmatory evidence will be provided from the long-term follow up of patients already treated in the study.

Along with the Biologics License Application approval, a Rare Disease Priority Review Voucher was granted. The company plans to monetise the voucher.