AlveoGene wins RPDD from FDA for AVG-002

Oxford, UK-based rare respiratory diseases focused AlveoGene today revealed it has been granted a Rare Pediatric Disease designation (RPDD) by the US Food and Drug Administration (FDA) for AVG-002, its novel, inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency.

The company explained that inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with very limited treatment options. This genetic disorder, affecting around 1 in 1 million newborns in the USA and Europe, results from mutations in the SP-B gene, which is essential for lung function and survival.

Current treatments are mainly supportive, including mechanical ventilation and surfactant replacement, however these interventions only provide temporary relief and once genetic diagnosis of SP-B deficiency is confirmed, treatment is usually withdrawn and patients die. The only definitive treatment, lung transplantation, is often not accessible owing to the scarcity of donor organs for newborns and the risks involved, leaving few if any viable options for long-term survival.