Welcome to the September 2025 edition of FDA Focus, a quick recap of some of the top FDA approvals of the month. Additionally, we'll take a look at the October pipeline and the potential approvals from the federal agency.
What you need to know:
- Arcutis submitted an sNDA for roflumilast cream 0.3% to treat plaque psoriasis in children as young as 2; “investigational Zoryve cream could be an important first-line treatment option,” said Adelaide Hebert, MD.
- FDA approved recombinant VWF for pediatric von Willebrand disease, the first non–plasma-derived therapy for this population; Vinay Prasad, MD, MPH, called it “a commitment to advancing treatment options for rare disorders.”
- FDA expanded ruxolitinib (Opzelura) to children aged 2–11 with atopic dermatitis; John Browning, MD, said, “It’s just so great to finally be able to get a 2A non-steroidal medicine as first line.”
Arcutis submits sNDA for roflumilast 0.3% for plaque psoriasis
Let's start on September 3, when Arcutis Biotherapeutics submitted a supplemental new drug application (sNDA) for the 0.3% formulation of roflumilast cream for the treatment of plaque psoriasis in children as young as 2 years.
If approved, the therapy would become the first phosphodiesterase 4 inhibitor available for this population.
Adelaide Hebert, MD, professor and chief of pediatric dermatology at University of Texas Health Houston, said, "There is a significant unmet need for nonsteroidal options that can effectively treat plaque psoriasis over the long term. If approved, investigational Zoryve cream could be an important first-line treatment option for children as young as age 2.”
Click here for more on the sNDA submission of roflumilast cream 0.3%.
FDA approves recombinant VWF for pediatric von Willebrand disease
On September 5, the FDA expanded the indication of recombinant von Willebrand factor (VWF) to include children with von Willebrand disease, to treat acute bleeding episodes, and for surgical use. The decision made recombinant VWF the first non–plasma-derived therapy available for this population in the United States, according to the FDA.2
The FDA's director of the Center for Biologics Evaluation and Research, Vinay Prasad, MD, MPH, said at the time of decision, "This approval highlights the FDA’s commitment to advancing treatment options for rare disorders, including for pediatric patients. When we see the combination of a strong biologic rationale, sound pharmacology, and meaningful clinical evidence, we are able to act quickly, even when studies involve smaller patient groups."
Click here for more on this FDA approval.
FDA approves ruxolitinib for pediatric patients with atopic dermatitis aged 2 to 11 years
On September 18, the federal agency approved an expanded indication for Incyte's ruxolitinib (Opzelura) to treat atopic dermatitis in patients aged 2 to 11 years. This adds to the previous indication of 12 years or older, which John Browning, chief of dermatology at Children's Hospital of San Antonio, is happy to see.3
"I think it is very exciting to have another nonsteroidal approved for kids with atopic dermatitis. [Ruxolitinib] has been approved in kids aged 12 years and older for vitiligo for some time now, so I do think that it is good to have multiple products approved that allow multiple options in treating atopic dermatitis."
"It's just so great to finally be able to get a 2A nonsteroidal medicine as first line, which is what I think it should be," he said. "I don't think we should have to make patients try a topical steroid first and [potentially] have a rebound issue.... It is just good to finally be able to start with these."
Click here for more on the FDA approval of ruxolitinib for pediatric atopic dermatitis.
Potential FDA approval of roflumilast cream 0.05% for pediatric atopic dermatitis
Keeping it in dermatology, as we look into October, we'll be watching for the potential FDA approval of topical roflumilast cream 0.05% for AD in children aged 2 to 5 years with mild to moderate atopic dermatitis.4
NDA submission was based on findings from multiple studies, including a phase 3 trial that involved over 650 children with a mean AD body surface area of 22%. Trial investigator Mercedes Gonzalez, MD, medical director of Pediatric Skin Research LLC, stated, "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."
Click here for more on roflumilast cream, 0.05%.
Thank you for watching September's FDA Focus. For ContemporaryPediatrics.com, I'm senior editor Joshua Fitch.
Editor's note: Stock images and videos above are courtesy of Adobe Stock.
References
- Ebert M. Arcutis submits sNDA for roflumilast cream 0.3% for treatment of psoriasis in children 2-5 years. Contemporary Pediatrics. September 3, 2025. Accessed October 3, 2025. https://www.contemporarypediatrics.com/view/arcutis-submits-snda-for-roflumilast-cream-0-3-for-treatment-of-psoriasis-in-children-2-5-years
- Campbell P. FDA approves first recombinant VWF for pediatric von Willebrand disease. Contemporary Pediatrics. September 5, 2025. Accessed October 3, 2025. https://www.contemporarypediatrics.com/view/fda-approves-first-recombinant-vwf-for-pediatric-von-willebrand-disease
- Fitch J. FDA approves ruxolitinib for pediatric patients with atopic dermatitis aged 2 to 11 years. Contemporary Pediatrics. September 18, 2025. Accessed October 3, 2025. https://www.contemporarypediatrics.com/view/fda-approves-ruxolitinib-for-pediatric-atopic-dermatitis
- Fitch J. FDA accepts roflumilast cream 0.05% sNDA for children with atopic dermatitis. Contemporary Pediatrics. February 26, 2025. Accessed October 3, 2025. https://www.contemporarypediatrics.com/view/fda-accepts-roflumilast-cream-0-05-snda-in-children-with-atopic-dermatitis
