Drug Pipeline News

In phase 3 SHINE-2, 50% of mirikizumab induction responders aged 6 to 17 with moderate to severe UC were in clinical remission at week 52.

Keisha L. Gibson, MD, MPH, discusses the FDA approval of Gazyva for childhood-onset INS and its implications for relapse prevention and pediatric care.

Topical botanical Cinainu earned FDA Breakthrough Therapy status for AA in patients aged 2+, supported by phase 2/3 RAAINBOW data in children.

The FDA accepted the Anaphylm NDA resubmission as a class 2 response, setting a March 16, 2027, PDUFA date for the sublingual epinephrine film.

Diana F. Clarke, PharmD, explains how IMPAACT 2023 supported dolutegravir from birth, plus dosing tips and open questions for preterm infants.

New ecopipam analyses show tic reduction within 8 weeks and sustained to 18 months in Tourette syndrome as the FDA weighs approval.

Oral icotrokinra sustained scalp, genital, and hand/foot psoriasis clearance through week 112 in ICONIC-TOTAL, with no new safety signals.

A recap of the top clinical trial and pipeline data readouts affecting pediatric care during September 2026.

Upadacitinib showed continued facial and body repigmentation in NSV through week 76, with numerically higher responses when combined with NB-UVB.

A recap of the top FDA approvals/regulatory decisions/pipeline updates affecting pediatric care during September 2026.

Joseph Rossano, MD, discusses how FDA approval of mavacamten introduces a disease-targeted treatment option for adults and pediatric patients weighing 30 kg (66 lbs) or more with symptomatic oHCM.

Mavacamten becomes the first cardiac myosin inhibitor approved for adults and pediatric patients weighing 30 kg (66 lbs) or more with symptomatic oHCM.

In a 48-patient phase 1b trial, ecnoglutide was well tolerated in adolescents with obesity and reduced BMI up to 12.6% at 20 weeks.

DBV filed a BLA for the Viaskin peanut patch in peanut-allergic children aged 4-7, citing phase 3 VITESSE data, and requested priority review.

ScolimiR-3D, a 6-microRNA blood test to predict severe curve progression in AIS, received FDA Breakthrough Device designation.

The FDA approved tiratricol (Emcitate) for peripheral thyrotoxicosis in MCT8 deficiency, the first approved therapy for the rare disorder.

FDA accepts leniolisib sNDA with priority review for children with APDS aged 4+ weighing 13 to <27 kg; PDUFA date is January 30, 2027.

FDA approves oral ALK2 inhibitor zilurgisertib for FOP in patients 12+, based on phase 2 PROGRESS data on new heterotopic ossification volume.

FDA approved obinutuzumab for relapsing or steroid-dependent childhood-onset INS, based on INShore data showing 95.5% vs 73.2% sustained remission.

Glucommander Pediatrics received FDA Breakthrough Device Designation for IV insulin dosing in hospitalized children younger than 2 years, but remains uncleared.

Lavengratinib increased annualized height velocity in 7 children with achondroplasia, but open-label phase 2 findings remain preliminary.

The FDA granted satralizumab priority review for MOGAD after phase 3 findings showed a 68% reduction in relapse risk versus placebo.

Amélie Lothe, PhD, discusses findings showing early, sustained benefits of fenfluramine treatment for patients with Lennox-Gastaut syndrome.

The FDA accepted the dersimelagon NDA with priority review for EPP and XLP; a decision on the oral MC1R agonist is expected by February 2027.

FDA approved levacetylleucine (Aqneursa) for ataxia in ataxia-telangiectasia, the first therapy for the disease, in patients 15 kg or heavier.