Drug Pipeline News

With the new drug application accepted with priority review, the FDA has assigned a date of November 20, 2025, to complete its review for potential approval.

The hyperpolarized contrast agent for oral inhalation can now be used in patients 6 years and older, lowering the approved indication from 12 years of age.

The decision makes hydrocortisone oral solution (Khindivi; Eton Pharmaceuticals) the only FDA-approved oral formulation of hydrocortisone.

Review some of the reactions from experts about the recent FDA approval of roflumilast foam 0.3% to treat scalp and body psoriasis in patients aged 12 years or older.

The approved indication now includes children down to 6 weeks of age to protect against invasive meningococcal disease via serogroups A, C, W, and Y.

Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.

The once-daily, steroid-free topical is now approved to treat plaque psoriasis of the scalp and body in patients aged 12 years and older.

The expanded indication follows original approval in August 2018 for use in previously-treated patients 12 years and older.

Get caught up with Contemporary Pediatrics! This list helps you navigate our top stories from the week, all in one place.

NVX-CoV2705 is indicated for individuals aged 12 to 64 years with an underlying condition that poses high risk for severe COVID-19 outcomes.

Delandistrogene moxeparvovec-rokl (ELEVIDYS) showed significant motor function improvements in 8- to 9-year-old Duchenne muscular dystrophy patients in part 2 of the phase 3 EMBARK study.

Somapacitan was non-inferior and had a similar safety profile and clinical outcomes compared to once-daily somatropin in improving yearly growth rates.

Safety, phosphate level gains, and immunogenicity data offer encouraging signals in the pivotal trial.

The federal agency provided BioCryst Pharmaceuticals a target action date of September 12, 2025 for potential approval of berotralstat (ORLADEYO).

This FDA decision will help to improve clarity and diagnostic accuracy of echocardiograms in pediatric patients, according to GE HealthCare.

The in vivo hematopoietic stem cell-directed therapy received rare pediatric disease and orphan drug designations earlier this year.

Subgroup data show crinecerfont helps children with CAH reduce steroid use while maintaining or improving androstenedione levels across diverse patient groups.

NEC is a life-threatening condition marked by the death of intestinal tissue, which impacts preterm infants.

Prescriptions for the epinephrine nasal spray are now available for patients aged 4 years and up who weigh 33 to < 66 lbs.

Nirsevimab significantly reduces RSV-related hospitalizations, ICU admissions, and LRTIs in infants, but doesn’t shorten hospital stays, according to a recent study.

A look back at the FDA submissions and regulatory decisions in the pediatric health care space from April 2025.

Stealth BioTherapeutics' CEO stated, "We hope to gain more information on the revised action date in the coming days."

The human FcRn-blocking monoclonal antibody is indicated for gMG patients aged 12 years and older.

The decision makes pz-cel the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with RDEB.

In addition, data presented at PAS revealed a predictable pharmacokinetic profile in the newborn population, consistent with adult humans.

From the FDA approvals of dupilumab and maralixibat tablets to a new puzzler case study, get caught up with our weekly review article.

The CRL issued by the FDA to Regeneron for extended EYLEA HD dosing to 24 weeks cited no safety or efficacy concerns.

The sBLA for dupilumab (Dupixent) was supported by data from the phase 3 LIBERTY-CUPID clinical program in patients with uncontrolled CSU.

Diazepam nasal spray (Valtoco) is approved for short-term treatment of seizure clusters, now in patients aged 2 years and older.

In the United States, maralixibat (Livmarli; Mirum Pharmaceuticals) is approved to treat both ALGS and PFIC, now in liquid and tablet formulations.