Eli Lilly and Company announced on September 25, 2025, that the U.S. Food and Drug Administration (FDA) has approved Inluriyo (imlunestrant), a 200 mg oral tablet, for the treatment of adults with estrogen receptor-positive (ER+), human epidermal growth factor receptor 2-negative (HER2-), ESR1-mutated advanced or metastatic breast cancer (MBC). This approval targets patients whose disease has progressed following at least one line of endocrine therapy (ET), addressing a critical need as approximately 50% of ER+, HER2- MBC patients develop ESR1 mutations after aromatase inhibitor exposure, leading to treatment resistance.
On September 25, 2025, the U.S. Food and Drug Administration (FDA) announced the approval of leucovorin calcium tablets for the treatment of cerebral folate deficiency (CFD), a neurological disorder characterized by impaired folate transport into the brain, resulting in developmental delays, autistic features (e.g., social communication challenges, sensory processing issues, repetitive behaviors), seizures, and motor coordination difficulties.
On September 22, 2025, the U.S. Food and Drug Administration (FDA) announced it is initiating a label change for acetaminophen, commonly known as Tylenol, to reflect evidence suggesting a potential association between its use during pregnancy and increased risks of autism spectrum disorder (ASD) and attention-deficit/hyperactivity disorder (ADHD) in offspring.
On September 19, 2025, the U.S. Food and Drug Administration (FDA) approved Merck’s KEYTRUDA QLEX™ (pembrolizumab and berahyaluronidase alfa-pmph) injection for subcutaneous use in adults across 38 solid tumor indications previously approved for KEYTRUDA® (pembrolizumab), marking it as the first subcutaneously administered immune checkpoint inhibitor.
Ionis Pharmaceuticals, Inc. received FDA approval on August 21, 2025, for DAWNZERA™ (donidalorsen), the first RNA-targeted therapy for prophylaxis against hereditary angioedema (HAE) attacks in adults and pediatric patients aged 12 and older. HAE, a rare genetic condition affecting approximately 7,000 Americans, causes recurrent, potentially life-threatening swelling in various body parts. DAWNZERA targets plasma prekallikrein (PKK), a key protein in HAE attack pathways, and is administered via subcutaneous autoinjector every four (Q4W) or eight weeks (Q8W), offering the longest dosing interval among HAE prophylactics. This approval marks Ionis’ second independent product launch within nine months, following TRYNGOLZA® for familial chylomicronemia syndrome.
Insmed Incorporated announced the U.S. Food and Drug Administration (FDA) approval of BRINSUPRI™ (brensocatib 10 mg and 25 mg tablets), the first approved treatment for non-cystic fibrosis bronchiectasis (NCFB) in adults and children aged 12 and older. As a first-in-class dipeptidyl peptidase 1 (DPP1) inhibitor, BRINSUPRI targets neutrophilic inflammation by inhibiting neutrophil serine proteases, addressing a root cause of airway damage, persistent infections, and exacerbations in NCFB. This chronic condition affects approximately 500,000 diagnosed patients in the U.S. and millions globally, characterized by permanently dilated bronchi, chronic cough, excessive mucus, shortness of breath, and frequent flares that worsen lung function and quality of life.
