FDA grants rare pediatric disease designation to neonatal lung therapy

The FDA has granted rare pediatric disease designation to vitamin A metabolic and reparative respiratory drugs manufactured by Advent Therapeutics to stop the development of bronchopulmonary dysplasia, according to a press release.

According to the release, these drugs are designed for use in infants born preterm and come in two forms: injectable and noninvasive inhaled neonatal lung therapies.

The FDA’s rare pediatric disease designation is granted to therapies designed to treat serious or life-threatening diseases that mainly occur among children and affect fewer than 200,000 people in the U.S.

If the FDA approves a new drug application for the vitamin A drugs, Advent Therapeutics may be able to receive a priority review voucher, which can be redeemed to obtain priority review for a future marketing application. Advent Therapeutics can also opt to sell or transfer the voucher.

“We are acutely focused on [bronchopulmonary dysplasia] as an area of high unmet medical need that presently has no FDA-approved therapies for prevention or treatment,” Dave L. Lopez, CEO of Advent Therapeutics, said in the release. “The FDA rare pediatric disease designation is an important addition to the orphan drug designations previously granted by the U.S. FDA and the European EMA for our vitamin A metabolic and reparative respiratory drugs.”

According to the release, Advent Therapeutics hopes to determine the next steps toward approval during an FDA prescription drug user fee act meeting planned for late 2023.