Wiskott-Aldrich syndrome (WAS) — disease overview, therapies, and what Waskyra changes
1) What is Wiskott-Aldrich syndrome (WAS) and how it is caused WAS is an X-linked primary immunodeficiency that typically affects males and is caused by mutations in the WAS gene, which encodes the Wiskott-Aldrich syndrome protein (WASp). WASp is expressed in hematopoietic cells and is essential for reorganization of the actin cytoskeleton during immune-cell activation, […]
FDA Approves First Interchangeable Biosimilar to Perjeta (pertuzumab) Genentech
On November 13, 2025, the FDA approved Poherdy (pertuzumab-dpzb, by Shanghai Henlius Biologics Co., Ltd.) as an interchangeable biosimilar to Perjeta (pertuzumab, by Genentech Inc.). U.S. Food and Drug Administration This marks the first biosimilar approved for Perjeta. U.S. Food and Drug Administration Indications for Poherdy include: The approval rests on comprehensive data comparing product […]
A Deep Dive into the FDA Approval of NDA 215244 (s000): What We Know, What It Means, and What Comes Next
Introduction The issuance of label 215244s000 marks the formal U.S. FDA approval of a new therapeutic agent (or new indication) under NDA 215,244. While publicly available summaries are scarce, the full prescribing label document offers a wealth of technical information that can be unpacked to understand the drug’s mechanism, safety/efficacy profile, clinical trial evidence, potential […]
RYBREVANT (amivantamab-vmjw) 2025 Label: What Changed, Why It Matters, and How to Apply It in Practice
Bottom line: In early 2025 the RYBREVANT label tightened safety language (notably infusion-related reactions, anaphylaxis placement, and venous thromboembolism management with lazertinib) and clarified dosing/administration details. Later in 2025, FDA accepted a supplement to update overall survival results from MARIPOSA, which will flow into Clinical Studies once the updated PI posts. Together, these moves round […]
Breaking News: FDA Expands IZERVAY™ Label for Long-Term Use in Geographic Atrophy Management
1. What’s New? The U.S. Food and Drug Administration has approved an expanded prescribing label for IZERVAY™, enabling unlimited duration dosing in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) Astellas Pharma US, Inc. | News Room. Previously, treatment duration was capped at 12 months. 2. Clinical Evidence Behind the Expansion This […]
EPYSQLI® (Eculizumab-aagh) — A Landmark Biosimilar in Rare Disease Treatment
Introduction Samsung Bioepis’ EPYSQLI® (eculizumab-aagh) has earned FDA approval as a biosimilar to Soliris® (eculizumab). Approved on July 19, 2024, EPYSQLI offers a more affordable alternative for treating debilitating rare diseases.FDA Access DataSamsung BioepisU.S. Pharmacist Indications EPYSQLI is indicated for: Efficacy & Clinical Performance EPYSQLI matches the reference biologic in both efficacy and safety: Cost […]
Byetta (Exenatide): A Leap Forward in Type 2 Diabetes Management
Key Details from the FDA Record (NDA 021773) Indication & Efficacy Mechanism of Action Byetta is a GLP-1 receptor agonist. It: Patent & Exclusivity Insights
EMA CHMP Spotlight: July 21–24, 2025 Meeting Highlights
1. New Medicines Recommended for Approval The EMA’s Committee for Medicinal Products for Human Use (CHMP) reviewed promising therapies and issued positive opinions for 13 new medicines, including treatments targeting rare, life-threatening, or underserved conditions.European Medicines Agency (EMA) Notable approvals: 2. Biosimilars & Generics: Expanding Access The CHMP also issued positive opinions for four biosimilars […]
Qfitlia (fitusiran): a practical, evidence-based look at the newly approved hemophilia prophylaxis
Summary: On March 28, 2025 the U.S. FDA approved Qfitlia (fitusiran) for routine prophylaxis to prevent or reduce bleeding in people 12 years and older with hemophilia A or B — with or without factor inhibitors. Qfitlia is an antithrombin-lowering, subcutaneously administered therapy that aims to restore thrombin generation rather than replacing missing clotting factors. […]