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Advent Therapeutics Granted FDA Rare Pediatric Disease Designation for Retinol Palmitate in Premature Infants' Bronchopulmonary Dysplasia Prevention

Friday, July 21, 2023

Advent Therapeutics, Inc., a late-stage biotechnology company specializing in retinol palmitate (vitamin A) drug products for neonatal lung therapies, has announced that it has been granted the U.S. Food and Drug Administration (FDA) Rare Pediatric Disease Designation (RPDD) for its vitamin A metabolic and reparative respiratory drugs aimed at preventing Bronchopulmonary Dysplasia (BPD) in premature infants.

The FDA's Rare Pediatric Disease Designation and Priority Voucher Program is intended to facilitate the development of drugs for rare pediatric diseases. Companies with an approved New Drug Application (NDA) for a rare pediatric disease may be eligible to receive a voucher, which can be used to prioritize the review of a subsequent marketing application for a different product or sold to another sponsor for their marketing application's priority review. This initiative encourages the development of novel drugs and biologics to treat rare pediatric diseases.

Dave L. Lopez, CEO of Advent Therapeutics, emphasized the urgency of addressing BPD, a condition with significant unmet medical needs and no FDA-approved therapies for prevention or treatment. The FDA Rare Pediatric Disease Designation adds to the Orphan Drug designations previously granted for Advent Therapeutics' vitamin A metabolic and reparative respiratory drugs by the US FDA and the European EMA.

Bronchopulmonary Dysplasia is a rare respiratory disorder that poses a critical threat to premature infants, leading to substantial morbidity and mortality. Currently, there are no approved drugs worldwide for BPD. Approximately 100,000 premature infants are at risk for BPD annually in the US and Europe, with many more affected in Asia-Pacific, Latin America, and other regions globally. The costs associated with treating BPD infants are exceptionally high, with an average hospital stay of 103 days and an average cost of $442,000 for treatment in the first year in the US. The overall healthcare expenses for treating BPD babies have been estimated to approach $2.5 billion annually in the US alone.

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