5 pediatric FDA decisions to watch for in Q4 2025 | Image credit: Contemporary Pediatrics

The fourth quarter of 2025 has all but arrived, and with it, several key potential approvals from the FDA for the pediatric population. From treatments for achondroplasia, to atopic dermatitis (AD) in children 2 to 5 years, these potential FDA decisions could influence treatment landscapes for children and teenagers.

With several decisions expected in the coming months, this short list below highlights what we're keeping a close eye on related to the remaining FDA pipeline for 2025.

Key FDA decisions to watch in Q4 2025

Roflumilast cream, 0.05% (Zoryve; Arcutis)

PDUFA: October 13, 2025

Indication: Atopic dermatits

Summary:

On February 26, 2025, Arcutis Biotherapeutics announced the FDA accepted a new drug application (NDA) for topical roflumilast cream 0.05% for AD in children aged 2 to 5 years. The 0.05% formulation of the once-daily, next generation phosphodiesterase-4 (PDE4) inhibitor is being evaluated for patients with mild-to-moderate AD.¹

NDA submission was based on data from multiple studies, including the INTEGUMENT-PED (NCT04845620) phase 3 trial involving 652 children with a mean AD Body Surface Area (BSA) of 22%.

"The data highlight the efficacy of the cream, along with its favorable safety and tolerability profile, which are critical when prescribing a long-term treatment for children with AD,” Mercedes E. Gonzalez, MD, medical director of Pediatric Skin Research, LLC, and INTEGUMENT-PED clinical trial investigator, said in a statement.

Click here for more on the NDA acceptance on roflumilast cream, 0.05%, as well as additional comments.

Navepegritide (TransCon CNP; Ascendis Pharma A/S)

PDUFA: November 30, 2025

Indication: Achondroplasia

Summary:

Ascendis Pharma's navepegritide has a decision set for November 30, 2025, to treat children with achondroplasia. The investigational prodrug of C-type natriuretic peptide (CNP) is administered once weekly and designed to treat individuals living with achondroplasia by providing continuous exposure of active CNP to receptor tissues across the body, such as growth plates and skeletal muscle.²

"[Navepegritide] is designed to provide sustained exposure to CNP, resulting in continuous inhibition of the FGFR3 pathway that is overactive in achondroplasia," said Janet Legare, MD, professor of Pediatrics, University of Wisconsin School of Medicine and Public Health, in a statement. "As a practicing physician, I am encouraged to see the FDA designating priority review for [navepegritide] as a potential new treatment option for children with achondroplasia.”

The federal agency accepted the NDA on June 2, 2025.

Click here for more on navepegritide.

Gepotidacin (Blujepa; GSK)

PDUFA: December 11, 2025

Indication:

Summary:

The FDA is scheduled to make a decision on GSK's gepotidacin (Blujepa) as an oral treatment option for uncomplicated urogenital gonorrhea in patients aged 12 years and older weighing at least 45 kg, with a PDUFA date of December 11, 2025.³

The sNDA is supported by findings from the phase 3 EAGLE-1 trial, published in The Lancet, which evaluated gepotidacin as a non-inferior oral alternative to the current leading treatment combination of intramuscular ceftriaxone and oral azithromycin. EAGLE-1 enrolled participants aged 12 years and older with suspected uncomplicated urogenital gonorrhea. Participants were randomly assigned to receive either gepotidacin (2 oral doses of 3000 mg administered 10–12 hours apart) or ceftriaxone (500 mg intramuscular) plus azithromycin (1 g oral).

Click here for more details and study data.

Lerodalcibep

PDUFA: December 12, 2025

Indication: Reduction of LDL-C

Summary:

On February 4, 2025, the FDA accepted a Biologics License Application (BLA) for LIB Therapeutics' lerodalcibep to reduce low-density lipoprotein cholesterol (LDL-C) for the treatment of patients with atherosclerotic cardiovascular disease (ASCVD), or very high or high risk of ASCVD, and primary hyperlipidemia, including heterozygous, and those 10 years or older with homozygous familial hypercholesterolemia (HeFH / HoFH).⁴

Lerodalcibep is a novel, small protein-binding, third-generation PCSK9 inhibitor, and has been developed as a more convenient, once-monthly, single small-volume, subcutaneous injection that will not require refrigeration at home or in travel, according to LIB.

Zoliflodacin

PDUFA: December 15, 2025

Indication: Uncomplicated gonorrhea

Summary:

On June 12, 2025, the FDA granted priority review for Innoviva Specialty Therapeutics' zoliflodacin to treat uncomplicated gonorrhea in adults and pediatric patients 12 years and older, with an assigned PDUFA date of December 15, 2025.⁵

Zoliflodacin is an investigational first-in-class antibacterial that is administered in a single oral dose for the treatment of uncomplicated gonorrhea.

According to Innoviva, there are more than 82 million new gonorrhea infectious globally each year, and is the second most common bacterial sexually transmitted infection.

References:

1. Fitch J. FDA accepts roflumilast cream 0.05% sNDA for children with atopic dermatitis. Contemporary Pediatrics. February 26, 2025. Accessed September 30, 2025. https://www.contemporarypediatrics.com/view/fda-accepts-roflumilast-cream-0-05-snda-in-children-with-atopic-dermatitis
2. Fitch J. FDA accepts navepegritide application to treat children with achondroplasia. Contemporary Pediatrics. June 2, 2025. Accessed September 30, 2025. https://www.contemporarypediatrics.com/view/fda-accepts-navepegritide-application-to-treat-children-with-achondroplasia
3. Ebert M. FDA accepts priority review for gepotidacin as oral treatment for gonorrhea. Contemporary Pediatrics. August 12, 2025. Accessed September 30, 2025. https://www.contemporarypediatrics.com/view/fda-accepts-priority-review-for-gepotidacin-as-oral-treatment-for-gonorrhea
4. LIB Therapeutics Announces FDA Acceptance of Biologics License Application for Lerodalcibep to Lower LDL-Cholesterol Across Broad Patient Population. LIB Therapeutics. Press release. February 10, 2025. Accessed September 30, 2025. https://www.biospace.com/press-releases/lib-therapeutics-announces-fda-acceptance-of-biologics-license-application-for-lerodalcibep-to-lower-ldl-cholesterol-across-broad-patient-population
5. FDA Grants Priority Review for Zoliflodacin New Drug Application for the Treatment of Uncomplicated Gonorrhea and Assigns Target PDUFA Date of December 15, 2025. Innoviva. Press release. June 12, 2025. Accessed September 30, 2025. https://investor.inva.com/news-releases/news-release-details/fda-grants-priority-review-zoliflodacin-new-drug-application