The U.S. Food and Drug Administration (FDA) granted Accelerated Approval to FORZINITY (elamipretide) on September 19, 2025, marking a pivotal moment as the first approved treatment for Barth Syndrome (BTHS) in eligible patients. This rare, X-linked, infantile-onset disease previously relied…
In a significant development for oncology practice, the U.S. Food and Drug Administration (FDA) on September 19, 2025, granted approval for Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph), a new subcutaneous formulation of the widely used PD-1 inhibitor, pembrolizumab. This approval…
On August 12, 2025, the U.S. Food and Drug Administration (FDA) approved Brinsupri (brensocatib), a novel, first-in-class dipeptidyl peptidase 1 (DPP1) inhibitor for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in patients aged 12 and older. This approval marks a…
The recent FDA approval of WAYRILZ (rilzabrutinib) marks a significant advancement for adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to previous treatments. Developed by Genzyme, a Sanofi company, this novel Bruton’s tyrosine kinase…
If your formulation plant is an asset, WHO-GMP vs USFDA compliance is its operating manual. Choosing the wrong compliance focus (or delaying upgrades) isn’t just a budget issue — it’s a market access and reputational risk that can cost far…
DAWNZERA (donidalorsen) is an FDA-approved antisense oligonucleotide indicated for prophylaxis of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older. Approved on 2025-08-21, DAWNZERA targets prekallikrein mRNA, reducing bradykinin production, the key mediator of angioedema attacks. In…
HER2-mutated non-small cell lung cancer (NSCLC) is a rapidly emerging segment, garnering intense interest from oncologists, patients, and pharma industry innovators. With the FDA’s accelerated approval of Hernexeos zongertinib, the landscape for HER2-driven NSCLC has fundamentally shifted—ushering in new hope for…
On August 6, 2025, the U.S. Food and Drug Administration (FDA) granted accelerated approval to MODEYSO (dordaviprone), a first-in-class protease activator for adult and pediatric patients aged one year and older with H3 K27M-mutant diffuse midline glioma (DMG) that has…
On 2025-07, the U.S. Food and Drug Administration (FDA) approved VIZZ aceclidine ophthalmic solution 1.44%, a first-in-class topical therapy for presbyopia [1]. Developed by LENZ Therapeutics, Inc., VIZZ is a novel cholinergic muscarinic agonist that offers a non-surgical, pharmacological approach…
SEPHIENCE (sepiapterin) is a novel phenylalanine hydroxylase (PAH) activator that has received approval for treating hyperphenylalaninemia (HPA) in adult and pediatric patients (1 month and older) with sepiapterin-responsive Phenylketonuria (PKU). Patients take SEPHIENCE as a once-daily oral powder with food…