Drug Pipeline News

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.

A review of the clinical potential of PTC Therapeutics' sepiapterin as a novel treatment for phenylketonuria (PKU).

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.

“Data from the Phase 3 ICONIC LEAD subgroup analysis demonstrate impressive efficacy rates, showing the promise of this novel therapeutic option," said Lawrence Eichenfield, MD.

The federal agency greenlit nivolumab with ipilimumab for treating MSI-H/dMMR colorectal cancer in adults and children 12 years and up.

If approved, the expanded indication would include children and adolescents aged 6 to 17 years who weigh 99 lbs (45 kg) or more.

View our Q1 2025 recap of standout pediatric news from FDA regulatory updates, clinical trial results, and expert commentary.