Drug Pipeline News

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.

The FDA cleared a generic Infuvite Pediatric injection for children receiving parenteral nutrition, adding a new option for hospital supply.

FDA clearance is being sought for roflumilast cream for infants 3 months and older with mild to moderate atopic dermatitis.

The FDA approved lunsotogene parvec-cwha as the first gene therapy for genetic hearing loss caused by biallelic OTOF variants.

The FDA approved dupilumab for children aged 2 to 11 years with chronic spontaneous urticaria, making it the first biologic for young pediatric CSU patients.

FDA expands teplizumab to children aged ≥1 year with stage 2 T1D, enabling earlier intervention to delay progression to insulin dependence.

FDA expands teplizumab (Tzeld) use to children as young as 1 year with stage 2 type 1 diabetes, aiming to delay progression to clinical disease.

The FDA has approved landiolol for patients younger than 18 years with supraventricular tachycardia, expanding access.

The FDA will review a supplemental application for delgocitinib cream in patients aged 12 to 17 years, supported by phase 3 data showing improved outcomes.

FDA fully approves sparsentan for FSGS in children 8 years and older, offering the first targeted option and stronger proteinuria drops than irbesartan.

A review of 6 novel drugs approved by the FDA, with links to coverage from Contemporary Pediatrics and other MJH Life Sciences brands.