FDA Grants RPDD to AlveoGene for AVG-002, Inhaled Gene Therapy for Lethal Neonatal SP-B Deficiency

AlveoGene, a company dedicated to improving outcomes for rare respiratory diseases through inhaled gene therapy, announced that the US Food & Drug Administration (FDA) has granted a Rare Pediatric Disease Designation (RPDD) to AVG-002, its innovative inhaled gene therapy targeting neonatal Surfactant Protein B (SP-B) deficiency. SP-B deficiency is a rare genetic disorder causing fatal respiratory distress in newborns, with an incidence rate of approximately 1 in 1 million live births in the US and Europe. The disorder, resulting from mutations in the SP-B gene, is currently addressed through supportive treatments such as mechanical ventilation and surfactant replacement, which only provide temporary relief. The only definitive treatment available is lung transplantation, which is not always feasible due to the scarcity of suitable donor organs and associated risks.

AVG-002, developed using AlveoGene’s proprietary InGenuiTy® platform, employs a unique pseudotyped lentiviral vector to efficiently deliver a functional SP-B gene to the deep lung alveolar region. Preclinical studies in SP-B gene knock-out murine models have shown that a single dose of AVG-002 significantly extends survival compared to other candidates in development, suggesting the potential for lifelong treatment from a single administration. These promising results are further supported by evidence of restored normal lung histology and function following AVG-002 treatment in disease-induced lung tissues. AlveoGene is currently advancing preparations for the clinical development of AVG-002, with the aim of filing for marketing authorization by 2028.

David Hipkiss, Executive Chair of AlveoGene, emphasized the importance of the RPDD for AVG-002, highlighting the urgent need for new therapies in this field and the transformative potential of this approach for treating SP-B deficiency in newborns. He also mentioned the company's expectations for AVG-003, another program targeting ABCA-3 deficiency—a rare pediatric genetic respiratory disorder that is 3-5 times more common than SP-B deficiency. AVG-003 utilizes the same InGenuiTy® platform, and AlveoGene is optimistic that it may also qualify for an RPDD as development progresses.

Additionally, AlveoGene is making significant progress with AVG-001, aimed at patients with lung disease caused by Alpha-1 Antitrypsin Deficiency (AATD). This candidate is designed to facilitate localized production of alpha-1 antitrypsin in the lungs, potentially offering a best-in-class treatment that directly targets the lung where it has the greatest impact on morbidity and mortality.

Rare pediatric diseases, as defined by the FDA, are serious or life-threatening conditions that primarily affect children under 18 and have fewer than 200,000 cases in the US. Companies awarded an RPDD receive a Priority Review Voucher (PRV) from the FDA upon the drug's approval for the designated pediatric indication. This voucher allows the recipient company to obtain FDA priority review for a different product, reducing review time by at least four months. PRVs can also be sold to other companies, with recent sales prices ranging from $100-$150 million.

David Hipkiss will provide an overview of AlveoGene and its technology at the upcoming Inv€$tival Showcase on 19 November in London, UK.

AlveoGene, founded in 2023 by Oxford Science Enterprises, Harrington Discovery Institute, and Old College Capital, in collaboration with leading scientists from the UK Respiratory Gene Therapy Consortium, aims to revolutionize the treatment of respiratory diseases through innovative inhaled gene therapies. The company’s pipeline includes AVG-002 and AVG-003 for SP-B and ABCA-3 deficiencies, respectively, and AVG-001 for Alpha-1 Antitrypsin Deficiency lung disease.