20 articles
Avlayah (tividenofusp alfa-eknm) becomes the first therapy approved to address brain damage in Hunter syndrome, a rare pediatric disorder.
Avlayah becomes the first treatment targeting neurologic symptoms of rare genetic disorder affecting 500 Americans.
Two breakthrough approvals target Hunter syndrome brain complications and cerebral folate deficiency for the first time.
Wellcovorin becomes the first approved treatment for cerebral folate deficiency tied to FOLR1 gene variants, offering hope for a rare neurological condition.
Baxdrostat becomes the first-in-class aldosterone synthase inhibitor approved to treat resistant hypertension alongside existing drugs.
FDA approves an updated Spinraza regimen for spinal muscular atrophy, targeting improved CSF drug levels for better neurological outcomes.
From Alzheimer's to Parkinson's, a wave of novel FDA approvals is expanding treatment options for serious neurological conditions.
A roundup of recent FDA brain-health approvals including lecanemab's full Alzheimer's approval and a new glioma drug.
Myqorzo (aficamten) wins FDA approval for symptomatic obstructive HCM, offering a novel cardiac myosin inhibitor option for patients.
The FDA expanded accelerated approval for zongertinib in HER2-mutant NSCLC, marking a targeted therapy milestone for a hard-to-treat mutation.
Zongertinib and sevabertinib earn FDA accelerated approvals for HER2-mutant NSCLC, expanding targeted therapy options for a historically hard-to-treat subset.
Sevabertinib and zongertinib win FDA approval for HER2-mutant NSCLC, expanding targeted therapy options for previously treated patients.
Zongertinib (Hernexeos) wins accelerated FDA approval for metastatic NSCLC with HER2 tyrosine kinase domain mutations, filling a critical treatment gap.
Zongertinib (Hernexeos) wins FDA accelerated approval for HER2-driven non-small cell lung cancer, offering a targeted oral option.
Tebipenem pivoxil becomes the first oral carbapenem approved in the US, offering a non-IV option for serious UTIs.
The FDA approved zenocutuzumab-zbco for adults with advanced, unresectable, or metastatic cholangiocarcinoma, a rare and aggressive bile duct cancer with limited treatment options.
New targeted therapy offers hope for patients with specific genetic mutations in non-small cell lung cancer.
Zongertinib (Hernexeos) wins FDA approval for unresectable or metastatic HER2-mutant non-squamous NSCLC, expanding targeted therapy options.
Sonrotoclax (Beqalzi), a next-gen BCL-2 inhibitor, wins accelerated FDA approval for relapsed or refractory MCL after BTK inhibitor failure.
Inqovi gains approval for newly diagnosed AML in patients 75+ or with comorbidities, offering a gentler oral treatment route.
