Drug Pipeline News

Watch our FDA pipeline news recap for the month of September 2025 as well as a preview of a key PDUFA date in October.

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

From another potential treatment for pediatric atopic dermatitis, to STI therapy for uncomplicated urogenital gonorrhea, take a look ahead at key PDUFA dates in Q4.

FDA approves guselkumab as the first IL-23 inhibitor for children 6 years or older with plaque psoriasis or psoriatic arthritis.

The approval expands on the previous indication of children 5 years and older with HoFH, approved in 2023.

Thomas Crawford, MD, discusses recent data for apitegromab for SMA, as well as overall developments in the field.

The FDA has authorized Essilor Stellest eyeglass lenses to correct myopia and slow progression in children, offering a lower-risk option than contacts.

Thomas Crawford, MD, said SAPPHIRE trial data show apitegromab provides clinically meaningful strength gains for children with SMA by targeting muscle in addition to motor neurons.

Observations were not specific to apitegromab, and the CRL did not cite any other approvability concerns related to efficacy and safety data.

The FDA announces significant updates on acetaminophen risks in pregnancy and approves leucovorin calcium for treating CFD in children.

Zilganersen met the primary endpoint of gait stabilization, showing statistically significant benefit on gait speed and favorable safety.

The planned phase 2 study of SAT-3247 will evaluate safety, tolerability, and functional outcomes in children with Duchenne muscular dystrophy.

The federal agency granted accelerated approval to elamipretide HCl to treat patients with Barth syndrome.

Rezpegaldesleukin improved disease severity and patient-reported outcomes in a phase 2b atopic dermatitis trial with sustained benefit.

The Phase 3 SURPASS-PEDS trial found tirzepatide improved glycemic control and weight outcomes in youth-onset type 2 diabetes, with benefits lasting one year.

In this video interview, John Browning, MD, comments on the recent pediatric indication approved for ruxolitinib to treat atopic dermatitis.

New phase 3 data show icotrokinra maintained high PASI90 responses in pediatric and adult patients at 1 year.

The updated label is based on real-world data collected since B-VEC was launched in the United States, following its approval in May 2023.

According to new, real-world data for trofinetide, caregivers reported quality-of-life, alertness, and interaction improvements at 12 months of treatment.

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

The FDA approves recombinant VWF for pediatric von Willebrand disease, enhancing treatment options for children during bleeding episodes and surgeries.

Watch our FDA pipeline news recap for the month for August 2025, as well as a preview of PDUFA dates listed for September.

Arcutis has submitted an sNDA to the FDA for roflumilast cream 0.3% for plaque psoriasis in children aged 2 to 5 years.

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

In recent studies, donidalorsen (Dawnzera) has demonstrated durable efficacy and long-term disease control.