Drug Pipeline News

The human, pasteurized human milk fortifier is approved for term infants (> 37 weeks) following corrective surgery for the gastrointestinal disorder gastroschisis.

Updated data from the phase 1/2 CHORD trial in 12 children with profound genetic hearing loss were presented at the Association for Research in Otolaryngology’s 48th Annual MidWinter Meeting.

Nearly 40% of children treated with roflumilast cream 0.05% achieved a 75% improvement in EASI-75.

The FDA has accepted PTC Therapeutics' NDA for vatiquinone, a potential treatment for Friedreich's ataxia, with a decision expected by August 19, 2025.

90-day interim biopsy data in the first 3 patients revealed an average microdystrophin expression of 110% and improvements in muscle health and resilience biomarkers.

Nurse practitioner Donna Hallas, PhD, PPCNP-BC, CPNP, PMHS, FAANP, FAAN offers her thoughts on the recent FDA approval of GSK's meningococcal ABCWY vaccine.

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

The combination vaccine targets 5 groups of the bacteria Neisseria meningitidis (A,B,C,W, AND Y) that cause the most invasive meningococcal disease cases (IMD) globally.

The federal agency has approved Merilog (insulin-aspart-szjj) as biosimilar to Novolog (insulin aspart) for the improvement of glycemic control in adults and pediatric patients with diabetes mellitus.

Ensoma's lead program, EN-374, has been granted a pair of FDA designations for the treatment of X-linked chronic granulomatous disease.

The new risdiplam tablet is suitable for those aged 2 years and older who weigh more than 44 lbs, according to Genentech.

Efficacy for the kinase inhibitor was based on the ReNue (NCT03962543 ) trial that featured 58 adults and 56 pediatric patients.

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

The investigational biologic targets the interleukin-2 receptor complex in the body in order to stimulate proliferation of inhibitory immune cells.

Cyclo Therapeutics has enrolled 10 patients in a single-arm sub-study treating newborns to 3 years of age, evaluating the Trappsol Cyclo in the youngest subsets.

The designations follow the FDA acceptance of IND application for ABO-101 to treat PH1, with a phase 1/2 study planned in the first half of 2025.

A recap of the FDA submissions and regulatory decisions in pediatrics from January 2025.

The primary and all key secondary endpoints were met compared to cream vehicle in the phase 3 DELTA TEEN trial.

Application submission is based on positive phase 3 data that demonstrated improvement in patients with type 2 and 3 SMA receiving current standard of care.

The higher dose regimen of nusinersen comprises a more rapid loading regimen, two 50 mg doses 14 days apart, and higher maintenance regimen, 28 mg, every 4 months.

The digestive enzyme cartridge is designed to mimic pancreatic lipase function.

The lead clinical trial investigator for a phase 3 study of crinecerfont in pediatric CAH patients breaks down the December 2024 FDA approval.

Neffy was first approved in September 2024 to treat type 1 allergic reactions in patients who weigh at least 66 lbs (33 kg).

Positive interim data was reported from the ENERGY 1 trial and Expanded Access Program that featured a total of 5 infants and 1 child.