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.
A randomized, double-blind, placebo-controlled trial, published in JAMA, evaluated metformin’s efficacy for knee osteoarthritis (OA) in 108 overweight or obese adults (BMI ≥25 kg/m², mean age ~60 years) with symptomatic knee OA (Kellgren-Lawrence grade 2-3). Conducted over 18 months, patients received metformin (up to 2g daily) or placebo alongside standard care. Primary outcomes were changes in knee pain (WOMAC pain score, 0-20 scale) and cartilage volume loss (via MRI). Secondary outcomes included physical function (WOMAC function score), weight loss, and safety.
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.
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.
Boehringer Ingelheim’s HERNEXEOS® (zongertinib tablets) has received accelerated approval from the U.S. Food and Drug Administration (FDA) as the first orally administered targeted therapy for adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) harboring HER2 (ERBB2) tyrosine kinase domain activating mutations, detected by an FDA-approved test, who have received prior systemic therapy.
