Drug Pipeline News

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.

The once daily extended-release oral suspension with nighttime dosing is approved to treat ADHD as a monotherapy or as an adjunctive therapy to approved CNS stimulant medications.

Approximately 40% of patients who receive PN in the United States as an intravenous source of nourishment are under 18 years of age.

The federal agency has accepted the sBLA for Priority Review designation, with a target action date of September 15, 2024.

The extended label approval is based on full evidence from the XTEND-Kids study.

Data from the ongoing phase 1/2 CHORD trial was presented at the ASGCT annual conference.

The new data, published in The Lancet, showed that nirsevimab substantially reduced RSV hospitalizations.

The resubmission was announced in a first quarter, 2024 earnings news release from Lilly, which expects "regulatory action in the second half of 2024."

The rare genetic disease causes the body's immune system to not function properly.

A decision from the federal agency is expected in the fourth quarter of 2024.