Q1 2025: A recap of the top headlines and interviews

Pediatric FDA, clinical trial, and interview recap from Q1 2025 | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

As we move into the second quarter of 2025, Q1 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 Q1 recap, Contemporary Pediatrics highlights some of the top FDA-related action items, clinical trial data, and expert interviews conducted during the first 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!

Q1 2025 regulatory updates in pediatrics

FDA approves diazoxide choline extended-release tablets for hyperphagia in Prader-Willi syndrome

The FDA approved diazoxide choline extended-release tablets (VYKAT XR; Soleno Therapeutics) on March 26, 2025, for treating hyperphagia in individuals aged 4 years and older with Prader-Willi syndrome (PWS). Granted priority review, breakthrough therapy, fast track, and orphan drug designations, VYKAT XR is the first approved treatment specifically addressing hyperphagia, a hallmark symptom of PWS.

In phase 3 trials, the treatment significantly improved hyperphagia, aggressive behaviors, fat mass, and metabolic parameters, with patients showing greater reductions in hyperphagia scores at 26 and 52 weeks compared to natural history data (P < 0.001). PWS, which affects approximately 1 in 15,000 live births, is associated with persistent hunger, cognitive disabilities, and metabolic challenges. Advocacy groups and researchers hailed the approval as a critical advancement for the PWS community.

FDA approves neffy 1 mg for anaphylaxis in children aged 4 years and older

The FDA approved epinephrine nasal spray (neffy; ARS Pharmaceuticals) 1 mg on March 5, 2025, for children aged 4 years and older weighing 33 to 66 lbs, making it the first needle-free epinephrine treatment for younger pediatric patients. This expands its indication beyond the original August 2024 approval for patients weighing at least 66 lbs.

Expected to launch in the United States by May 2025, neffy offers a user-friendly, temperature-stable alternative to traditional epinephrine auto-injectors, addressing fears of needle-based administration that often delay treatment. Approval was based on pharmacokinetic and pharmacodynamic data showing comparable efficacy to injectable epinephrine.

“The availability of a needle-free epinephrine option for children is a breakthrough in the treatment of severe allergic reactions,” said David Fleischer, MD, section head of Allergy & Immunology and professor of Pediatrics at Children's Hospital Colorado, in a statement.

FDA accepts new drug application for vatiquinone to treat Friedreich's ataxia

The FDA has accepted PTC Therapeutics' new drug application (NDA) for vatiquinone to treat Friedreich's ataxia (FA), granting it priority review with a target action date of August 19, 2025. If approved, vatiquinone would be the first therapy for pediatric FA patients and a potential treatment option for adults.

The NDA is supported by data from the MOVE-FA trial and long-term studies, demonstrating that vatiquinone slows disease progression and is well tolerated. The drug inhibits 15-Lipoxygenase (15-LO) to mitigate mitochondrial dysfunction and oxidative stress, key factors in FA. Clinical trial results showed benefits in fatigue reduction, neurological symptoms, and mobility preservation.

FA is a rare genetic neuromuscular disorder affecting approximately 25,000 people worldwide, with no approved treatment targeting disease progression.

Apitegromab BLA submitted to FDA for spinal muscular atrophy

On January 29, 2025, Scholar Rock submitted a biologics license application (BLA) to the FDA for apitegromab, a muscle-targeted therapy for spinal muscular atrophy (SMA), supported by data from the phase 3 SAPPHIRE and phase 2 TOPAZ trials.

The SAPPHIRE trial met its primary endpoint, demonstrating a statistically significant 1.8-point improvement in the Hammersmith Functional Motor Scale-Expanded (HFMSE) at 52 weeks for patients receiving apitegromab compared to placebo. Secondary endpoints suggested additional motor benefits beyond SMN-targeted therapy alone. Apitegromab was well tolerated, with no serious treatment-related adverse events. Scholar Rock has requested priority review, which, if granted, would shorten the FDA’s review timeline.

FDA approves GSK's meningococcal ABCWY vaccine

On February 14, 2025, the FDA approved the meningococcal ABCWY (MenABCWY, Penmenvy; GSK) vaccine for individuals aged 10 to 25 years, offering protection against the five major serogroups (A, B, C, W, Y) of Neisseria meningitidis, which cause invasive meningococcal disease (IMD).

The vaccine combines components of the meningococcal group B vaccine (Bexsero) and the ACYW conjugate vaccine (Menveo). Approval was based on a phase 3 trial (NCT04502693) involving 3,650 participants across seven countries, demonstrating strong immunogenicity, non-inferiority to existing vaccines, and a well-tolerated safety profile. MenB accounts for over half of IMD cases in Sadolescents and young adults, yet vaccination rates remain low.

Q1 2025 trial announcements in pediatrics

Phase 3: Delgocitinib improves chronic hand eczema in adolescents

ON February 4, 2025, LEO Pharma A/S announced positive phase 3 results for delgocitinib (Anzupgo) 20 mg/g cream in adolescents aged 12 to 17 years with chronic hand eczema (CHE), meeting all primary and key secondary endpoints in the DELTA TEEN trial (NCT05355818). Delgocitinib, a topical pan-Janus kinase (JAK) inhibitor, is already approved for adults in several markets but currently has no approved options for adolescents with moderate to severe CHE.

The trial demonstrated significant improvement based on the Investigator’s Global Assessment for CHE treatment success at Week 16. CHE is a persistent form of hand eczema that affects quality of life, with symptoms such as itch, pain, and scaling. LEO Pharma plans to present full trial data in scientific forums, highlighting the treatment’s potential for an underserved adolescent population.

DB-OTO demonstrates improved hearing in 10 of 11 children with profound genetic hearing loss

On February 24, 2025, Regeneron Pharmaceuticals presented new phase 1/2 CHORD trial (NCT05788536) data on DB-OTO gene therapy at the Association for Research in Otolaryngology’s (ARO) 48th Annual MidWinter Meeting.

Of 11 children with profound genetic hearing loss who had at least one post-treatment assessment, 10 demonstrated hearing improvements. The first child dosed at 10 months showed speech and developmental progress at 72 weeks. DB-OTO targets otoferlin-related hearing loss, a rare condition affecting auditory nerve communication.

Results included near-normal hearing in some participants and positive auditory brainstem responses. No serious adverse events were reported. DB-OTO has received multiple FDA designations, including orphan drug and fast track status.

Roflumilast cream 0.05% demonstrates eczema area severity, itch improvement for pediatric atopic dermatitis

On February 24, 2025, Pediatric Dermatology published phase 3 INTEGUMENT-PED trial (NCT04845620) data showing that roflumilast cream 0.05% (Zoryve; Arcutis Biotherapeutics) improved eczema severity and itch in children aged 2 to 5 years with atopic dermatitis (AD).

At week 4, 25.4% of treated children achieved validated Investigator Global Assessment-Atopic Dermatitis (vIGA-AD) success vs. 10.7% with vehicle (P < 0.0001), and 39.4% reached EASI-75 improvement.

Rapid itch relief occurred within 24 hours (P ≤ .0014). Findings supported Arcutis’ December 2024 FDA submission for approval. If approved, roflumilast 0.05% would offer a steroid-free, once-daily treatment for young children with AD.

Omalizumab outperforms oral immunotherapy in treating multi-food allergy

On March 2, 2025, results from the OUtMATCH trial showed that omalizumab (Xolair; Genentech, Novartis) was more effective than oral immunotherapy (OIT) in treating multi-food allergy.

Among all participants, 36% of those receiving omalizumab tolerated at least 2g of peanut protein and two other allergens, compared to 19% in the OIT group. High rates of allergic reactions led to a 49% dropout rate in the OIT group, while none discontinued omalizumab due to severe reactions. Omalizumab, which reduces immune sensitivity by targeting IgE, may offer a well-tolerated alternative to OIT for food allergy treatment.

Cingulate reports phase 3 safety data for CTx-1301 to treat ADHD in children, adolescents

On March 4, 2025, Cingulate Inc. announced phase 3 safety data for CTx-1301 (dexmethylphenidate), a once-daily stimulant for ADHD designed to provide full-day symptom control.

Findings from two pediatric trials showed no serious adverse events, supporting the company’s planned FDA submission in mid-2025. CTx-1301 delivers three precisely timed doses in a single tablet, potentially addressing adherence issues and side effects linked to multiple daily doses.

Cingulate was scheduled to meet with the FDA on April 2, 2025, for a pre-NDA meeting, with a full submission expected in the summer of 2025.

Q1 2025 expert perspectives in pediatrics

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

RX Review: Advances in pediatric food allergy and anaphylaxis treatment

In this Contemporary Pediatrics and HCPLive RX Review video discussion, Brian Schroer, MD, an allergist-immunologist at Cleveland Clinic Children's Hospital, moderated a panel on the latest advancements in pediatric food allergy care. Joined by Colleen Kraft, MD, of Children's Hospital Los Angeles, and Russell Traister, MD, of Allegheny Health Network, the experts discussed the shift toward early allergen introduction, the growing role of oral immunotherapy and intranasal epinephrine, and the impact of Xolair (omalizumab) in treating food allergies. They highlighted the importance of personalized management plans to enhance diagnosis, treatment, and long-term patient outcomes.

Tina Tan, MD discusses the increased outbreaks of norovirus this year

In this video interview, Tina Tan, MD, FAAP, FIDSA, FPIDS, highlighted the alarming rise in norovirus outbreaks, with CDC data showing 495 outbreaks from August to December 2024.

Tan, an infectious diseases expert at Lurie Children's Hospital and president of the Infectious Diseases Society of America, explained that norovirus spreads easily through contaminated food, surfaces, and direct contact, particularly in schools and daycare centers.

Tan stressed the importance of proper handwashing—since hand sanitizers are ineffective—along with food safety measures to curb transmission. Pediatricians play a key role in educating families on hygiene practices to prevent infection.

Discussing FDA approved neffy with Russell Libby, MD, FAAP

In this Contemporary Pediatrics video interview, Russell Libby, MD, FAAP, founder of Virginia Pediatric Group, discusses the FDA approval of neffy, a needle-free epinephrine nasal spray for anaphylaxis.

Libby highlights its advantages over auto-injectors, including improved temperature stability, a longer shelf life of over two years, and easier administration during emergencies.

He emphasizes that neffy’s reliability and convenience could make it a preferred option for patients, provided it remains affordable and accessible through insurance formularies.