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

2023-07-24

孤儿药申请上市优先审批

Reinventing Vitamin A for neonatal and pediatric unmet medical needs

Bronchopulmonary Dysplasia (BPD) is most frequent serious complication of preterm birth – US preterm births highest in 15 years

Advent plans FDA PDUFA Meeting late-2023 to establish pathway for US approval of lead injectable product

Advent Therapeutics, Inc., a late-stage biotechnology company developing precisely optimized retinol palmitate (vitamin A) drug products as both injectable and breakthrough non-invasive inhaled neonatal lung therapies, announced today that it has received U.S. Food and Drug Administration (FDA) Rare Pediatric Disease Designation (RPDD) for its vitamin A metabolic and reparative respiratory drugs for the prevention of Bronchopulmonary Dysplasia (BPD) in premature infants.

FDA’s Rare Pediatric Disease Designation and Priority Voucher Program is intended to facilitate the development of new drugs for the prevention and treatment of rare pediatric diseases. Companies that receive approval for a New Drug Application (NDA) for a rare pediatric disease may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application. This program is intended to encourage the development of new drugs and biologics for the treatment of rare pediatric diseases.

“We are acutely focused on BPD as an area of high unmet medical need that presently has no FDA-approved therapies for prevention or treatment. The FDA Rare Pediatric Disease Designation is an important addition to the Orphan Drug designations previously granted by the US FDA and the European EMA for our vitamin A metabolic and reparative respiratory drugs,” said Dave L. Lopez, CEO of Advent Therapeutics.

ABOUT BPD

BPD is a rare disease and the most common serious complication facing premature infants, resulting in significant morbidity and mortality. It is an extremely costly respiratory disorder afflicting premature infants for which there is currently no approved drugs worldwide. Annually, in both US and Europe, there are approximately 100,000 premature infants at-risk for BPD and many more in Asia-Pacific, Latin America and throughout the world. In the US, hospital costs for treating a BPD infant in the first year average $442,000 with an average hospital stay of 103 days. US healthcare costs of treating babies with BPD have been estimated as approaching $2.5 billion per year.

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