Drug Pipeline News

The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, as the first treatment for pediatric MPS IIIA (Sanfilippo syndrome type A).

Aquestive resubmitted its NDA for dibutepinephrine sublingual film Anaphylm, citing new human factors and PK data addressing the FDA's CRL.

Phase 3 CROSSING trial data show tezepelumab improved histologic remission and dysphagia versus placebo through week 52 in EoE.

FDA approved apitegromab-mstn (Isembyld) for SMA in patients 2 years and up, based on phase 3 data showing added motor gains with SMN2 therapy.

XOCOVA gives pediatricians a new oral option to help prevent COVID-19 after exposure in adolescents aged 12 years and older.

Week 52 data from the reACHin trial show navepegritide stabilized foramen magnum stenosis and boosted growth in infants with achondroplasia.

Phase 3 CANOPY-HCH-3 data show vosoritide significantly improved growth velocity, height, and arm span in children with hypochondroplasia.

Semaglutide plus lifestyle changes moved 40.4% of children below the obesity threshold at 68 weeks in the phase 3 STEP Young trial.

A recap of the top FDA approvals, authorizations, and regulatory decisions affecting pediatric care during August 2026.

The FDA approved zilganersen (Zanvastro) for Alexander disease in patients from infancy through adulthood, based on Phase 1-3 gait speed data.

The FDA has approved Tivicay PD (dolutegravir) to treat HIV-1 in newborns from birth, based on IMPAACT 2023 pharmacokinetic and safety data.

FDA De Novo authorization establishes Avulux lenses as an option for migraine-associated light sensitivity in patients aged 12 years and older.

FDA authorized Abbott's Libre Duo 10 Day, the first wearable that continuously tracks glucose and ketones, for patients 2 and older with diabetes.

FDA approved nipocalimab (Imaavy) for wAIHA in patients 12 and older, based on Phase 2/3 ENERGY trial data on durable hemoglobin response.

Phase 3 data show lebrikizumab improved skin clearance, itch, and quality of life in children with moderate-to-severe atopic dermatitis.

Infliximab or steroids may be more effective than anakinra as adjunctive therapy for children with MIS-C who do not respond to IVIG.

FDA granted priority review to Teva's ecopipam for pediatric Tourette syndrome, backed by phase 3 data showing reduced relapse risk.

Scholar Rock says apitegromab's SMA BLA stays on track for its September 30, 2026, PDUFA date after dropping one of 2 fill-finish sites.

Phase 3 SMART data support the safety of clesrovimab in infants and children at increased risk for severe RSV disease across 2 seasons.

Revised Anaphylm packaging cut pouch-opening time from 17 to 3 seconds, and self-administered PK matched clinician-administered data.

Phase 3 SMART trial found clesrovimab was well tolerated in high-risk infants through 2 RSV seasons, with safety comparable to palivizumab.

FDA accepted Merck's sBLA extending ENFLONSIA (clesrovimab-cfor) to high-risk children under 2 in their second RSV season.

A look back at the FDA approvals, regulatory decisions, and pipeline updates in the pediatric health care space from July 2026.

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

CTx-1301 improved clinician-rated ADHD symptoms versus placebo in a phase 3 pediatric trial, but detailed safety data remain limited.