Drug Pipeline News

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.

Aquestive Therapeutics reports positive pediatric study results for Anaphylm, supporting its FDA submission for treating severe allergic reactions.

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

Submission is based on 3 clinical trials, including data from the ApproaCH Trial among children with achondroplasia.

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

Fitusiran is administered subcutaneously starting once every 2 months for patients aged 12 years and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors.

A look ahead to 5 key regulatory decisions scheduled to take place during the second quarter of 2025, from a monoclonal antibody to topical psoriasis treatments.

Mesoblast's remestemcel-L (Ryoncil) was approved by the FDA on December 18, 2024 to treat SR-aGVHD in patients 2 months and older. Now, it is available for purchase.

A new clinical trial has found that omalizumab (Xolair; Genetech, Novartis) is more effective than oral immunotherapy (OIT) in treating multi-food allergy in individuals with severe allergic reactions to small amounts of common food allergens.

Diazoxide choline has been granted breakthrough, fast track, and orphan drug designations in the United States.

In this Q+A interview, Craig McDonald, MD, discusses the interim, 90-day data from the phase 1/2 INSPIRE DUCHENNE trial, evaluating SGT-003, a gene therapy candidate for DMD.

The approval marks a vital step toward care for urinary tract infections, helping to reduce recurrence and improve patients’ quality of life.

Results were from the phase 3 STEER study among a broad population of patients with SMA aged 2 to under 18 years.

Prior to approval on February 14, 2025, the only FDA approved vaccine for chikungunya protection was indicated for 18 years and up.

In the final part of this 5-part series, panelists discuss new allergy treatments such as Xolair, early immunotherapy, and their broader impact.

In part 4 of this 5-part series, panelists discuss gaps in pediatric food allergy care, stressing better diagnosis, education, and management.

In part 3 of this 5-part series, panelists discuss the recent Neffy approval, oral immunotherapy, and Xolair’s role in allergy management.