Q2 2025: A recap of the top headlines and interviews

Q2 2025: A recap of the top headlines and interviews | Image Credit: © Uladzislau - stock.adobe.com.

As we move into the third quarter of 2025, Q2 brought several treatment regulatory updates and clinical trial results to help better take care of pediatric patients across the globe.

In the vast realm of pediatric health care, several specialty areas, from dermatology to allergy and immunology to infectious disease, saw newly FDA-approved medicines and updated clinical data for treatments still in development.

In the below Q2 recap, Contemporary Pediatrics highlights some of the top FDA-related action items, clinical trial data, and expert interviews conducted during the second quarter of 2025.

Click on each title below for additional information based on each news article.

To stay up to date on all aspects of pediatric health care, consider subscribing to our free e-newsletters, which contain regular headlines and clinical trial updates. Emails are sent 3 times per week on Tuesdays, Thursdays, and Saturdays.

Thank you for reading Contemporary Pediatrics!

Q2 2025 regulatory updates in pediatrics

FDA approves clesrovimab to protect infants during first RSV season

On June 9, 2025, the FDA approved Merck’s clesrovimab (Enflonsia), an extended half-life monoclonal antibody, to protect infants from respiratory syncytial virus (RSV) during their first RSV season. The approval was supported by positive results from the phase 2b/3 CLEVER and phase 3 SMART trials, which demonstrated that a single dose of clesrovimab significantly reduced RSV-related infections and hospitalizations in both healthy and high-risk infants. In the CLEVER trial, clesrovimab reduced medically attended lower respiratory infections by 60.5%, RSV-related hospitalizations by 84.3%, and severe RSV cases by over 90% through 5 months post-dose (P < 0.001 for all). Unlike other monoclonal antibodies, clesrovimab targets site 4 of the RSV fusion protein, neutralizing more than 96% of RSV A and B strains. The therapy, given as a fixed intramuscular dose, offers direct, rapid, and durable protection throughout a typical RSV season.

FDA approves twice-yearly lenacapavir as PrEP for HIV in adolescents, adults

On June 18, 2025, the FDA approved lenacapavir (Yeztugo; Gilead Sciences), the first and only twice-yearly injectable HIV-1 capsid inhibitor for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV in individuals aged 12 and older weighing at least 35 kg. Approval was based on results from the phase 3 PURPOSE 1 and PURPOSE 2 trials, in which lenacapavir showed no HIV infections among cisgender women in sub-Saharan Africa (PURPOSE 1) and just 2 infections among 2179 participants in a diverse population including cisgender men and gender-diverse people (PURPOSE 2), achieving over 99.9% effectiveness in both trials. The treatment demonstrated superiority over once-daily oral Truvada (F/TDF), offering a long-acting option for people who prefer or require less frequent dosing. Experts hailed the approval as a major step forward in HIV prevention, potentially improving adherence and reducing stigma associated with daily PrEP.

FDA accepts NDA for epinephrine sublingual film to treat type 1 allergic reactions

On June 17, 2025, the FDA accepted the new drug application for epinephrine sublingual film (Anaphylm; Aquestive Therapeutics), a potential first-in-class, orally delivered treatment for type 1 allergic reactions, including anaphylaxis. If approved by the January 31, 2026, target action date, Anaphylm would be the only device-free epinephrine option available in the United States. The thin, postage–stamp–sized film dissolves without water and is designed for portability and weather resistance. A recent pediatric study in children aged 7 to 17 years confirmed consistent pharmacokinetics with adult data and showed no serious adverse events. Experts emphasized the product’s potential to overcome key barriers to epinephrine use, including fear of injection and the inconvenience of carrying autoinjectors.

FDA approves garadacimab-gxii to prevent HAE attacks in patients aged 12 years, older

On June 17, 2025, FDA approved CSL’s garadacimab-gxii (Andembry), a first-in-class monoclonal antibody targeting factor XIIa, to prevent hereditary angioedema (HAE) attacks in patients aged 12 years and older. Approval was based on the phase 3 VANGUARD trial, where once-monthly garadacimab-gxii reduced mean HAE attack rates by 86.5% and median attack rates by over 99% compared to placebo (P < 0.001). An open-label extension study also showed sustained efficacy and a favorable long-term safety profile. Experts highlighted its novel mechanism and convenient dosing as key advances for HAE management. Meanwhile, FDA review of another HAE therapy, sebetralstat, has been delayed due to resource constraints, though no concerns about its safety or efficacy were raised.

FDA approves at-home belimumab autoinjector for children with lupus nephritis

On June 24, 2025, the FDA approved a 200 mg/mL autoinjector formulation of belimumab (Benlysta; GSK) for at-home use in children aged 5 years and older with active lupus nephritis (LN), making it the first subcutaneous, self-administered biologic treatment for pediatric LN. Originally approved for adult SLE in 2011 and expanded to pediatric indications in later years, this new formulation offers a convenient alternative to intravenous infusions, potentially reducing clinic visits and easing treatment burdens for families. The decision was based on belimumab’s established safety and efficacy profile. Experts noted that pediatric lupus is often more severe than adult-onset disease, making flexible treatment options critical. The autoinjector is now available for eligible patients and can be administered by a health care provider or trained caregiver at home.

Q2 2025 trial announcements in pediatrics

Bavarian Nordic initiates trial of chikungunya vaccine in children aged 2-11 years

On June 12, 2025, Bavarian Nordic initiated a phase 3 clinical trial of its single-dose, virus-like particle (VLP) chikungunya vaccine, Vimkunya, in children aged 2 to 11 years. The global, randomized, double-blind, placebo-controlled study will assess the vaccine’s safety and immunogenicity in 720 participants over 2 years, with primary results expected in 2028. Vimkunya, already FDA-approved for individuals aged 12 and older, was launched in March 2025 and is the first VLP chikungunya vaccine available for that age group. The pediatric trial reflects the company’s effort to expand access to protection amid increasing global transmission of chikungunya.

Phase 3 trial of omaveloxolone to treat Friedreich's ataxia in children aged 2 to <16 begins

On June 18, 2025, Biogen began dosing participants in the BRAVE study, a global phase 3 trial evaluating omaveloxolone (Skyclarys) in children aged 2 to under 16 years with Friedreich ataxia (FA). Currently approved for individuals 16 and older, this is the first time the drug is being studied in a younger pediatric population. The 52-week randomized, placebo-controlled trial will enroll approximately 255 children to assess safety, efficacy, and pharmacologic outcomes, with Upright Stability Score (USS) as the primary endpoint. Omaveloxolone was originally approved by the FDA in 2023 as the first treatment for FA and has demonstrated long-term functional benefits in previous trials. Biogen emphasized the urgent need for pediatric treatment options, given the rapid disease progression in early-onset FA, and plans to expand enrollment globally as regulatory approvals are secured.

First infant enrolled in trial of roflumilast cream, 0.05% for atopic dermatitis in infants

On June 10, 2025, Arcutis Biotherapeutics announced the enrollment of the first patient in INTEGUMENT-INFANT, a phase 2 open-label trial evaluating roflumilast cream 0.05% in infants aged 3 months to under 2 years with mild-to-moderate atopic dermatitis (AD). The study will assess safety and tolerability over 4 weeks in approximately 35 participants. Roflumilast, a topical PDE4 inhibitor, is already approved in various formulations for multiple dermatologic conditions in older children and adults. This trial represents a critical step toward expanding treatment options for the youngest patients, a group with limited approved therapies. Arcutis highlighted the potential of roflumilast to reduce steroid use while providing long-term relief, addressing the significant quality-of-life burden AD imposes on children and caregivers.

Dupilumab improves atopic dermatitis severity in adolescents, adults with skin of color

Announced June 9, 2025, the phase 4 DISCOVER trial, dupilumab (Dupixent; Regeneron and Sanofi) showed significant clinical benefits in patients aged 12 years and older with moderate-to-severe atopic dermatitis (AD) and skin of color. Among the 120 participants, 82% of whom were Black, 76% achieved at least a 75% improvement in disease severity (EASI-75) at 24 weeks, with many seeing results as early as 2 weeks. Dupilumab also improved itch in 53% of patients and reduced post-inflammatory hyperpigmentation by 53%. The trial is the first large-scale study of its kind in this population and highlights dupilumab’s potential to address both the physical and quality-of-life impacts of AD in underserved communities.

Six-month IXCHIQ chikungunya vaccine data demonstrates sustained antibody levels

On June 5, 2025, new data from the phase 2 VLA1553-221 trial showed that Valneva’s chikungunya vaccine, IXCHIQ (Valneva), provided strong antibody persistence and maintained a favorable safety profile up to 6 months post-vaccination in children aged 1 to 11 years. In the trial, a full dose elicited a more robust immune response than a half dose, with a 96.5% seroresponse rate at day 180 among chikungunya virus–naïve children. The vaccine was well tolerated across all dosing levels, with no safety concerns identified. These findings support full-dose use in pediatric populations and build on earlier adolescent and adult data. Valneva emphasized the importance of a single-dose solution for long-term protection, especially in low- and middle-income countries where vaccine access may be limited. IXCHIQ is currently FDA-approved for adults 18 and older at risk of chikungunya virus exposure.

Q2 2025 expert perspectives in pediatrics

In this section, click the "play" button on each video interview to watch.

Octavio Ramilo, MD, reacts to the FDA approval of clesrovimab for RSV prevention

In this video interview, Octavio Ramilo, MD, chair of Infectious Diseases at St. Jude Children’s Research Hospital, discussed the June 9, 2025, FDA approval of clesrovimab (Enflonsia; Merck) for preventing RSV disease in infants during their first season. Ramilo highlighted the significance of this advancement, noting that clesrovimab joins nirsevimab and Pfizer’s maternal RSV vaccine as new tools to reduce RSV-related hospitalizations. He explained clesrovimab’s unique mechanism of targeting site 4 of the RSV F protein, effective in both pre- and post-fusion forms, and emphasized the benefits of uniform dosing without weight adjustment. Ramilo underscored the importance of early protection and the potential long-term benefits of passive immunization in reducing future respiratory issues, such as wheezing or asthma.

RX Review: RSV prevention and pediatric hospitalization

In this HCP Live Network RX Review roundtable, Albert Rizzo, MD, of the American Lung Association, moderated a discussion on the evolving landscape of RSV prevention in pediatric care. He was joined by Tina Tan, MD, of Lurie Children's Hospital, and Joanne Nazif, MD, of Children’s Hospital at Montefiore. The panel explored the clinical impact of recent advancements such as maternal RSV vaccination and monoclonal antibody use. Tan noted reductions in RSV-related hospitalizations and disease severity in infants, particularly after the introduction of nirsevimab, while Nazif emphasized that treatment options remained limited, making prevention all the more critical. The panel also reviewed shifting RSV trends following the COVID-19 pandemic and highlighted early data from the 2024–2025 season showing declines in infant hospitalizations, suggesting improved outcomes through proactive prevention strategies.

Jennifer Soung, MD, reacts to the FDA approval of roflumilast foam 0.3%

In this video interview, Jennifer Soung, MD, director of clinical research at Southern California Dermatology, discussed the May 22, 2025, FDA approval of roflumilast (Zoryve; Arcutis Biotherapeutics) foam 0.3% for treating plaque psoriasis of the scalp and body in patients aged 12 and older. Soung highlighted the significance of having a highly effective nonsteroidal option, particularly for adolescents, citing long-standing demand from parents. She reviewed findings from the phase 3 ARRECTOR trial, where roflumilast showed superior improvements in both scalp and body psoriasis compared to vehicle, as well as rapid itch relief in some patients within 24 hours. Soung emphasized the psychosocial toll psoriasis takes on youth and praised the inclusion of adolescents in the clinical trials, noting that concurrent pediatric and adult study design helps ease treatment access for younger patients and their families.