FDA Grants Advent’s Therapy for Premature Infants Rare Pediatric Disease Designation

Rare Daily Staff

The U.S. Food and Drug Administration granted Advent Therapeutics Rare Pediatric Disease designation for its vitamin A metabolic and reparative respiratory drugs for the prevention of bronchopulmonary dysplasia in premature infants.

Bronchopulmonary dysplasia (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 United States 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 United States, hospital costs for treating a BPD infant in the first year averages $442,000 with an average hospital stay of 103 days. Total U.S. healthcare costs of treating babies with BPD have been estimated as approaching $2.5 billion per year.

Advent has designed proprietary water miscible vitamin A drug products for optimal administration to fragile preterm neonates. The drugs do not contain chlorobutanol (an alcohol) or other preservatives that may be of concern with respect to sensitivity or potential toxicity. Advent’s formulation of vitamin A comes in intramuscular and aerosolized forms for use in neonatal intensive care units as orphan drugs to address unmet medical needs such as BPD, retinopathy of prematurity, and acute lung injury.

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. The program is intended to encourage the development of new drugs and biologics for the treatment of rare pediatric diseases. A rare pediatric disease is defined as a serious or life-threatening condition affecting individuals younger than 18 years and with a prevalence under 200,000 people in the United States.

The designation is potentially lucrative because 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. Most recently, Sarepta Therapeutics sold a PRV for $102 million.

“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,” said Dave Lopez, CEO of Advent Therapeutics. “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.”