Landmark FDA Approvals Usher in New Era of Pediatric Healthcare in 2025
The first half of 2025 has witnessed a surge of groundbreaking FDA approvals poised to dramatically expand treatment options and improve access to care for infants, children, and adolescents facing a wide range of health challenges. From innovative vaccines and needle-free emergency treatments to therapies targeting rare diseases, HIV prevention, and autoimmune disorders, these decisions reflect significant strides in both medical innovation and accessibility. Many of the newly authorized therapies offer more convenient dosing schedules, at-home administration options, or represent first-in-class mechanisms, addressing long-standing clinical gaps and supporting increasingly personalized care.
A Wave of Innovation: Key Approvals Shaping Pediatric Care
Contemporary Pediatrics highlights the most impactful FDA decisions from January through June 2025 that are reshaping the future of care for young patients. These approvals signal a commitment to addressing unmet needs and improving the quality of life for children and their families.
Protecting Against Meningococcal Disease with GSK’s Novel Vaccine
On February 14, 2025, the FDA approved GSK’s meningococcal ABCWY vaccine (MenABCWY, Penmenvy) for individuals aged 10 to 25 years. This vaccine offers crucial protection against the five major serogroups (A, B, C, W, Y) of Neisseria meningitidis, the bacteria responsible for invasive meningococcal disease (IMD). The vaccine combines components of previously approved meningococcal group B (Bexsero) and ACYW conjugate (Menveo) vaccines.
A phase 3 trial (NCT04502693) involving 3,650 participants across seven countries demonstrated the vaccine’s strong immunogenicity, non-inferiority to existing vaccines, and a well-tolerated safety profile. Despite MenB accounting for over half of IMD cases in adolescents and young adults, vaccination rates remain concerningly low, underscoring the importance of this new preventative measure.
Needle-Free Epinephrine for Anaphylaxis: A Game Changer for Young Children
A significant advancement in emergency care arrived on March 5, 2025, with the FDA approval of neffy, a 1 mg epinephrine nasal spray (ARS Pharmaceuticals), for children aged 4 years and older weighing 33 to 66 lbs. This marks the first needle-free epinephrine treatment specifically approved for this younger pediatric population, expanding upon a previous approval for patients weighing at least 66 lbs issued in August 2024.
Expected to be available in the United States by May 2025, neffy provides a user-friendly, temperature-stable alternative to traditional epinephrine auto-injectors. According to a company release, this addresses a critical barrier to treatment – the fear of needles that often delays life-saving intervention. Data from pharmacokinetic and pharmacodynamic studies confirmed neffy’s comparable efficacy to injectable epinephrine.
Addressing Hyperphagia in Prader-Willi Syndrome with VYKAT XR
The FDA approved diazoxide choline extended-release tablets (VYKAT XR; Soleno Therapeutics) on March 26, 2025, for the treatment of hyperphagia – excessive eating – in individuals aged 4 years and older with Prader-Willi syndrome (PWS). VYKAT XR received priority review, breakthrough therapy, fast track, and orphan drug designations, highlighting the urgent need for effective treatments for this rare genetic disorder.
Phase 3 trials demonstrated that VYKAT XR significantly improved hyperphagia, aggressive behaviors, fat mass, and metabolic parameters. Patients showed greater reductions in hyperphagia scores at 26 and 52 weeks compared to natural history data (P < 0.001). PWS, affecting approximately 1 in 15,000 live births, is characterized by persistent hunger, cognitive disabilities, and metabolic challenges. Advocacy groups and researchers have hailed this approval as a critical advancement for the PWS community.
Merck’s Clesrovimab Offers Robust Protection Against RSV
On June 9, 2025, the FDA approved clesrovimab (Enflonsia; Merck), an extended half-life monoclonal antibody, to protect infants from respiratory syncytial virus (RSV) during their first RSV season. The approval was supported by compelling results from the phase 2b/3 CLEVER and phase 3 SMART trials, which demonstrated a single dose of clesrovimab significantly reduced RSV-related infections and hospitalizations in both healthy and high-risk infants.
The CLEVER trial showed 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). Notably, clesrovimab targets site 4 of the RSV fusion protein, neutralizing more than 96% of both RSV A and B strains.
Expanded Access to Ustekinumab-stba Biosimilar for Pediatric Psoriasis and Arthritis
The FDA approved a new presentation of ustekinumab-stba (Steqeyma; Celltrion), a biosimilar to ustekinumab (Stelara), on June 15, 2025. The new 45mg/0.5mL single-dose vial for subcutaneous injection is indicated for patients aged 6 to 17 years weighing under 60 kg with plaque psoriasis or psoriatic arthritis. This approval ensures that ustekinumab-stba now matches all dosing forms of the reference product. Phase 3 data confirmed clinical similarity to the reference biologic, with no meaningful differences in safety or efficacy.
Novel Therapy for Hereditary Angioedema: Garadacimab-gxii Approved
On June 17, 2025, the FDA approved garadacimab-gxii (Andembry; CSL), a first-in-class monoclonal antibody targeting factor XIIa, for the prevention of hereditary angioedema (HAE) attacks in patients aged 12 years and older. The phase 3 VANGUARD trial demonstrated that 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 further confirmed sustained efficacy and a favorable long-term safety profile. Experts highlighted its novel mechanism and convenient dosing as key advances in HAE management. However, FDA review of another HAE therapy, sebetralstat, has been delayed due to resource constraints, though no safety or efficacy concerns were raised.
Twice-Yearly HIV PrEP: Lenacapavir Offers New Hope for Adolescents and Adults
A major step forward in HIV prevention occurred on June 18, 2025, with the FDA approval of lenacapavir (Yeztugo; Gilead Sciences), the first and only twice-yearly injectable HIV-1 capsid inhibitor for pre-exposure prophylaxis (PrEP). The approval extends to individuals aged 12 and older weighing at least 35 kg, reducing the risk of sexually acquired HIV.
Results from the phase 3 PURPOSE 1 and PURPOSE 2 trials were highly encouraging, showing no HIV infections among cisgender women in sub-Saharan Africa (PURPOSE 1) and only 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. Lenacapavir demonstrated superiority over once-daily oral Truvada (F/TDF), offering a long-acting option that may improve adherence and reduce stigma.
At-Home Belimumab Autoinjector Approved for Pediatric 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). This represents the first subcutaneous, self-administered biologic treatment for pediatric LN. Originally approved for adult SLE in 2011 and subsequently expanded to pediatric indications, this new formulation offers a convenient alternative to intravenous infusions, potentially reducing clinic visits and easing the treatment burden for families. Experts note that pediatric lupus is often more severe than adult-onset disease, making flexible treatment options particularly critical.
These FDA approvals collectively represent a period of remarkable progress in pediatric healthcare, promising improved outcomes and a better quality of life for countless children and adolescents.
