Drug Pipeline News

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.

Expert recommendations outline how crinecerfont may help reduce glucocorticoid exposure in children with classic CAH.

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

Expert recommendations outline how crinecerfont may help reduce glucocorticoid exposure in children with classic CAH.

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

The FDA approved ocrelizumab for pediatric RRMS in patients 10 years and older after positive OPERETTA II trial results.

ABS-1230 entered phase 1b/2 testing for KCNT1-related epilepsy as Actio also joined the FDA Rare Disease Evidence Principles process.

Two-year data from the pivotal ApproaCH trial showed sustained growth improvements with TransCon CNP in pediatric achondroplasia.

FDA expands ASCENIV label to include pediatric patients aged ≥2 years with primary immunodeficiency, broadening earlier treatment access.

FDA granted rare pediatric disease designation to SB-101 for urea cycle disorders, a cell-based liver therapy set for phase 1/2 testing.

FDA approved insulin glargine-aldy as an interchangeable biosimilar to Lantus for type 1 diabetes and adult type 2 diabetes.

FDA granted rare pediatric disease designation to investigational (Z)-endoxifen for McCune-Albright syndrome in girls.

Phase 3 sunRIZE trial of ersodetug in congenital hyperinsulinism missed its primary endpoint but showed CGM-based glycemic gains.

Two-year CAHtalyst data show crinecerfont sustains androgen reduction and lowers glucocorticoid doses in children with congenital adrenal hyperplasia.

Phase 3 data show berdazimer gel improved lesion clearance vs vehicle in molluscum contagiosum with a favorable safety profile.

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

FDA cleared Earflo for children 2 years and older with negative middle ear pressure, expanding at-home autoinflation options.