Drug Pipeline News

The FDA has accepted Takeda's supplemental application for IV vedolizumab in patients ages 2 and older with moderate-to-severe UC or Crohn's disease, with a PDUFA date set for Q1 2027.

The FDA has approved an every-8-week maintenance regimen for lebrikizumab-lbkz (EBGLYSS), giving eligible adults and adolescents with moderate-to-severe atopic dermatitis as few as 6 injections per year without mandatory topical therapy.

The FDA has approved bemotrizinol as the first new active sunscreen ingredient under the OTC monograph in nearly two decades, clearing the broad-spectrum UV filter for use in adults and children 6 months of age and older.

FDA designations for investigational GEn-1123 may support development in Duchenne muscular dystrophy, but clinical data remain limited.

Vertex reported phase 3 ALYFTREK data in children aged 2 to 5 years with cystic fibrosis and responsive CFTR genotypes.

The FDA has broadened the approved indication for marstacimab (Hympavzi; Pfizer) to include pediatric patients ages 6–11 with hemophilia A or B and patients 12 and older with inhibitors.

A look back at the latest clinical trial updates for the pediatric population during May 2026.

FDA approved ensitrelvir for COVID-19 post-exposure prophylaxis in patients aged 12 years and older after positive phase 3 trial results.

A look back at the FDA submissions and regulatory decisions in the pediatric health care space from May 2026.

The FDA accepted an sNDA for mavacamten in adolescents with obstructive hypertrophic cardiomyopathy, with a decision expected in September 2026.

This decision marks the first FDA-approved inhaled insulin for pediatric patients.

The FDA extended its review of adrabetadex for infantile-onset Niemann-Pick disease type C to November 17, 2026, after a major NDA amendment.

FDA expanded approval of linaclotide for functional constipation to include children aged 2 to 5 years.

FDA approval moves adapalene 0.1% and benzoyl peroxide 2.5% gel to OTC use for acne in patients aged 12 years and older.

Ecopipam lowered relapse risk in pediatric Tourette syndrome responders in phase 3 data supporting a planned FDA submission.

Phase 3 data showed vosoritide significantly improved annualized growth velocity and height outcomes in children with hypochondroplasia.

FDA accepted a levacetylleucine sNDA for ataxia-telangiectasia and set a September 19, 2026, target action date.

Early phase 2 data suggest zovegalisib may reduce lesion volume and improve symptoms in PIK3CA-driven vascular anomalies.

Early OTC-HOPE data link ECUR-506 to fewer hyperammonemic events in infants with neonatal-onset OTC deficiency.

BMN 401 met a biochemical end point but missed radiographic improvement in a phase 3 trial of children with ENPP1 deficiency.

Preclinical data for Gemma's GB703 in DMD and GB221 in SMA1 highlight early gene therapy strategies, but clinical questions remain.

RGX-202 met a microdystrophin biomarker endpoint in phase 3 Duchenne muscular dystrophy data, with functional findings still preliminary.

A UK phase 2b trial will evaluate Lactin-V for preventing preterm birth in pregnant patients at high risk of early delivery.

RVL-001, a vorinostat formulation, has entered early placebo-controlled studies in Rett syndrome and Pitt-Hopkins syndrome.

Early AK-OTOF trial data showed hearing improvements and favorable safety in children with OTOF-mediated hearing loss.