Drug Pipeline News

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.

The FDA granted priority review to efzimfotase alfa for HPP in patients aged 2 years and older; a decision is expected in the first half of 2027.

Adolescent VALIANT subgroup data show pegcetacoplan cut proteinuria 75% vs placebo, supporting its expanded pediatric kidney indication.

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.