August's FDA Focus: Approvals and pipeline updates in pediatrics

Welcome to this month's FDA Focus, where we take you back and recap some of the top FDA approvals of the month, and provide a look ahead on what to expect in the month to come from the federal agency.

First up on August 5, the FDA approved fremanezumab-vfrm (Ajovy; Teva Pharmaceuticals), to prevent episodic migraine in children aged 6 years and older, who weigh at least 99 lbs. Previously indicated for adults, the drug is administered as a 225-mg monthly subcutaneous injection. Approval was based on data from the phase 3 SPACE trial, which demonstrated a significant reduction in monthly migraine days and a tolerability profile consistent with adult studies.¹

The next day, the agency approved tocilizumab-anoh for cytokine release syndrome (CRS) in patients 2 years and older, bringing this biosimilar into full indication alignment with its reference drug Actemra. The IL-6 receptor antagonist is also approved for patients with pJIA and sJIA aged 2 years and older, as well as other adult indications. According to Celltrion, tocolizumab-anoh was expected to be available in the US on August 31st.²

Brensocatib was approved on August 12th to become the first treatment for non-cystic fibrosis brochiectasis (NCFB) in patients 12 years and older. The treatment was evaluated in the phase 3 ASPEN trial, which featured more than 1700 patients. Annualized pulmonary exacerbation rates were significantly reduced compared to placebo. Both the 10- and 25-mg doses of brensocatib improved exacerbation-related outcomes, with the latter also having slowed lung function decline.³

The same day, the agency approved iron sucrose injection, USP as the first generic version of Venofer for the treatment of iron deficiency anemia in adults and children aged 2 years and older with chronic kidney disease. The IV product became the first generic version of Venofer injection.⁴

As we look into September, we are keeping watch for the potential approval of berotralstat for HAE in patients aged 2 to 11 years. The FDA assigned a target action date of September 12th, and previously granted priority review designation. If approved, berotralstat would become the first targeted oral prophylactic therapy for patients with HAE under the age of 12 years.⁵

Click here for more on berotralstat.

Additionally, we'll be watching for the potential approval of ruxolitinib cream 0.75% to treat mild-to-moderate atopic dermatitis in patients aged 2 to 11 years. Following an extension of its review period, the FDA assigned a new target action date for September 19th. The sNDA submission was supported by results from the phase 3 TRuE-AD3 study, which evaluated the safety and efficacy of ruxolitinib cream.⁶

The trial met its primary endpoint, with a significantly higher proportion of patients treated with ruxolitinib achieving Investigator’s Global Assessment-treatment success (IGA-TS) compared to those treated with vehicle cream.

The study also met a key secondary endpoint, showing that more patients in the treatment group achieved at least a 75% improvement in the Eczema Area and Severity Index (EASI75) at week 8, compared to those using the vehicle.

Thank you for watching August's FDA Focus. For Contemporary Pediatrics.com, I'm senior editor Joshua Fitch.

Editor's note: All images/videos above are courtesy of Adobe Stock.

References:

1. Fitch J. FDA approves fremanezumab-vfrm for migraine prevention in children aged 6 years, older. Contemporary Pediatrics. August 5, 2025. Accessed September 4, 2025. https://www.contemporarypediatrics.com/view/fda-approves-fremanezumab-vfrm-for-migraine-prevention-in-children-aged-6-years-older
2. Fitch J. FDA approves tocilizumab-anoh for CRS in patients 2 years and older. Contemporary Pediatrics. August 6, 2025. Accessed September 4, 2025. https://www.contemporarypediatrics.com/view/fda-approves-tocilizumab-anoh-for-crs-in-patients-2-years-and-older
3. Fitch J. FDA approves brensocatib to treat adolescents with non–cystic fibrosis bronchiectasis. Contemporary Pediatrics. August 12, 2025. Accessed September 4, 2025. https://www.contemporarypediatrics.com/view/fda-approves-brensocatib-to-treat-adolescents-with-non-cystic-fibrosis-bronchiectasis
4. Ebert M. FDA approves first generic iron sucrose injection for iron deficiency anemia in CKD. Contemporary Pediatrics. August 12, 2025. Accessed September 4, 2025. https://www.contemporarypediatrics.com/view/fda-approves-first-generic-iron-sucrose-injection-for-iron-deficiency-anemia-in-ckd
5. Fitch J. FDA accepts NDA for berotralstat in HAE patients aged 2 to 11 years. Contemporary Pediatrics. May 14, 2025. Accessed September 4, 2025. https://www.contemporarypediatrics.com/view/fda-accepts-nda-for-berotralstat-in-hea-patients-aged-2-to-11-years
6. Ebert M. FDA extends review of ruxolitinib cream for pediatric atopic dermatitis. Contemporary Pediatrics. June 20, 2025. Accessed September 4, 2025. https://www.contemporarypediatrics.com/view/fda-extends-review-of-ruxolitinib-cream-for-pediatric-atopic-dermatitis