According to AlveoGene, the FDA has granted orphan drug designation to the company’s AVG-002 inhaled gene therapy for the treatment of neonatal surfactant protein B deficiency. AVG-002 also recently received rare pediatric disease designation for that indication. Alveogene reiterated its plans to initiate clinical development with the goal of a regulatory submission as soon as 2028.
The company, which was launched in 2023, is developing inhaled gene therapies based on a lentiviral delivery platform developed by the UK Respiratory Gene Therapy Consortium. In addition to AVG-002, AlveoGene’s pipeline includes AVG-001 inhaled gene therapy for the treatment of alpha-1 antitrypsin deficiency and AVG-003 inhaled gene therapy for ABCA-3 deficiency.
AlveoGene Executive Chair David Hipkiss commented, “Receiving orphan drug designation from FDA for AVG-002 represents another important milestone for AlveoGene, following the recent grant of rare pediatric disease designation for the same candidate. Both awards highlight the significant unmet need for novel therapies targeting SP-B deficiency in newborn infants, which is invariably fatal within a few months and for which there are very limited treatment options. AVG-002, a novel gene therapy, has been designed using our InGenuiTy technology to reach deep into the affected lung tissues to restore lung function and could provide a lifelong treatment from a single administration. We are totally committed to advancing AVG-002 as quickly as possible to provide a transformative, life-saving treatment for patients.”
Read the AlveoGene press release.
