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."
