AlveoGene, a company dedicated to revolutionizing treatments for rare respiratory conditions through inhaled gene therapies, has announced that its novel therapy, AVG-002, has received Orphan Drug Designation from the US Food & Drug Administration (FDA). This recognition is for AVG-002’s potential to treat the deadly neonatal Surfactant Protein B (SP-B) deficiency. This status follows FDA’s recent Rare Pediatric Disease Designation granted to AVG-002 in November.
SP-B deficiency is a rare genetic disorder, found in approximately 1 in 1 million newborns in the US and Europe, leading to fatal respiratory distress syndrome with very few treatment options. This condition arises from mutations in the SP-B gene, critical for proper lung function and survival.
Currently, available treatments are predominantly supportive, such as mechanical ventilation and surfactant replacement, providing only short-lived relief. Upon genetic diagnosis of SP-B deficiency, treatments typically cease, resulting in patient fatalities. Lung transplantation, the only definitive treatment, is often not a viable choice due to the lack of donor organs and associated risks, leaving minimal options for survival.
David Hipkiss, AlveoGene’s executive chair, explained: “Securing Orphan Drug Designation from the FDA for AVG-002 marks another vital step forward for AlveoGene, after obtaining the Rare Pediatric Disease Designation for the same treatment. Both recognitions underscore the urgent need for groundbreaking therapies for SP-B deficiency in newborns, a condition generally fatal within months with scant treatment alternatives. AVG-002 is uniquely designed using our InGenuiTy technology to address lung dysfunction with a single application potentially offering lifelong treatment. We are unwavering in our pursuit to expedite AVG-002’s development to deliver a potentially life-saving treatment.”
AlveoGene is harnessing its InGenuiTy platform to develop AVG-002, using a novel pseudotyped lentiviral vector that delivers an active SP-B gene into the neonatal deep lung alveolar area effectively through respiratory instillation.
Preclinical results in SP-B gene knockout mouse models show that a single dose of AVG-002 significantly extends lifespan beyond what other investigational SP-B deficiency treatments have reported, suggesting a single administration could potentially result in a lifelong remedy. These promising results are bolstered by evidence of lung function and histological restoration post-AVG-002 treatment in disease-altered lung tissues. Encouraged by these findings, AlveoGene is progressing toward the clinical development phase of AVG-002 with the target of marketing authorization by 2028.
The FDA’s Orphan Drug Designation supports developments for medicines targeting rare diseases affecting fewer than 200,000 people in the US, offering incentives like tax credits, fee exemptions, and seven years of market exclusivity upon drug approval.
AlveoGene is a niche gene therapy company focused on lung-targeted therapies aimed at advancing respiratory disease treatment standards and providing solutions where existing options are limited or non-existent.
The UK Respiratory Gene Therapy Consortium (GTC) unites leading researchers from Imperial College London and the Universities of Oxford and Edinburgh, drawing on over two decades of collaborative expertise in gene therapy innovation for respiratory conditions.