AlveoGene grabs pediatric rare disease tag for inhaled gene therapy

AlveoGene has been granted a Rare Pediatric Disease Designation (RPDD) by the U.S. FDA for its novel inhaled gene therapy for the rare respiratory disease, neonatal Surfactant Protein B (SP-B) deficiency.

AVG-002 is being developed by AlveoGene using its proprietary InGenuiTy platform, which employs a unique pseudotyped lentiviral vector to deliver a functional SP-B gene directly to the neonatal deep lung alveolar region with high efficiency and efficacy via respiratory instillation.

Inherited SP-B deficiency is an ultra-rare monogenic cause of fatal respiratory distress syndrome in newborn infants with very limited treatment options. The genetic disorder 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only provide temporary relief and once genetic diagnosis of SP-B deficiency is confirmed, treatment is usually withdrawn and patients die.

According to AlveoGene, preclinical data in SP-B gene knock-out murine models demonstrate that a single dose of AVG-002 extends survival substantially longer when compared with reported data of other SP-B deficiency candidates in development, offering the possibility of a lifelong treatment from a single administration. These data are further reinforced by findings that confirm the restoration of normal lung histology and function following AVG-002 treatment in disease-induced lung tissues. 

Encouraged by the data, AlveoGene — which was launched in 2023 in partnership with leading scientists from the world-renowned UK Respiratory Gene Therapy Consortium — is advancing its preparations for the clinical development of AVG-002 in lethal neonatal SP-B deficiency with the possibility of filing for market authorization by 2028.

Companies awarded an RPDD receive a rare pediatric disease priority review voucher (PRV) from the FDA when the designated drug is approved for the associated indication in the pediatric population. The voucher allows the recipient company to obtain FDA priority NDA or BLA review for a different product and/or indication, which can speed market entry by at least four months. The PRV may be used by the original recipient or sold.