Drug Pipeline News

With the expanded label, Palforzia is now approved to treat individuals aged 1 to 17 years with a confirmed peanut allergy diagnosis, after the treatment was originally approved in 4-to-17-year-olds in January 2020.

ConSynance Therapeutics' CSTI-500, an innovative Triple Monoamine Reuptake Inhibitor, has received FDA's Rare Pediatric Disease Designation for treating Prader-Willi Syndrome in children.

From baseline to final assessment, all 7 matched cerliponase alfa-treated children under 3 years of age maintained a motor score of 3, representing a "grossly normal gait, signifying a delay in disease onset," stated BioMarin.

Initial approval for maralixibat was granted on March 13, 2024, to treat PFIC patients aged 5 years and older.

Merck announced clesrovimab met all primary safety and efficacy endpoints, with additional detailed findings to be presented at an upcoming scientific congress.

With the acceptance of the BLA, a new PDUFA date of January 7, 2025 has been set.

If approved, roflumilast foam 0.3% for scalp and body psoriasis treatment would add to the multiple already-approved roflumilast indications.

The Rare Pediatric Disease Designation from the FDA follows the previously granted Orphan Drug and Fast Track Designations.

Get caught up with our journal! Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

Details of newly FDA-approved roflumilast cream 0.15% for AD in pediatric patients, plus insight and commentary from Lawrence Eichenfield, MD.

An intentional launch date for ustekinumab-ttwe in the United States is set for sometime in February 2025.

The decision target action date was July 7, 2024. At this time, there is no indication from the federal agency to extend the date further.

Get caught up with our journal! Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

Results confirmed that a bedaquiline-containing regimen offered a significant improvement in treatment outcomes compared to injectable-containing regimens.

FDA decisions for the 2 accepted NDAs have target action dates of December 29 and December 30, 2024, respectively.

The submission to the FDA includes data from the phase 2b ReNeu trial of patients aged 2 years and older with NF1-associated PN causing significant morbidity.

Get caught up with our journal! Review some of the top stories from the Contemporary Pediatrics website over the last week, and catch up on anything you may have missed.

New data for FDA-approved dupilumab (Dupixent; Regeneron and Sanofi) highlights positive study results for the eosinophilic esophagitis (EoE) treatment in children aged 1 to 11 years.

The decision makes pitolisant the first and only FDA-approved, non-scheduled treatment option for daytime sleepiness in this patient population.

For ambulatory Duchenne patients, delandistrogene moxeparvovec-rokl was granted traditional approval, while accelerated approval was granted for non-ambulatory patients.

The company also announced that it has surpassed its interim analysis enrollment target and expects topline phase 3 data from the FORTIFY study (NCT05775848) among individuals with LGMD21/R9 in 2025.

The decision is based on statistical significance achieved in the T2NOW trial, which demonstrated reduction of A1C.

pJIA can lead to an increased risk of permanent joint damage as well as delayed growth and development.

Once-daily, steroid-free, topical roflumilast cream 0.15% has a PDUFA date of July 7, 2024.

The 2-drug, fixed-dose combination tablet contains darunavir, an HIV-1 protease inhibitor, and cobicistat, a CYP3A inhibitor that serves as a PK enhancer or “booster.”

Additionally, a new weight-based oral solution, RINVOQ LQ, is now available as an option for the pediatric populations.

Mesoblast Limited's chief executive announced a BLA resubmission for remestemcel-L is expected to be filed with the FDA this quarter.

The maximum daily dose has increased from 80 mg to 100 mg.

Prior to the federal agency's decision, children aged 5 years and older could only receive belimumab through an intravenous (IV) formulation.

The therapy was previously granted accelerated approval for the thyroid cancer indications in adults and pediatric patients aged 12 years and older.