hemophilia A

Jul 17, 2026

Summary H2 2026 is expected to be a highly active FDA regulatory period, with decisions spanning oncology, nephrology, infectious diseases, neurology, hematology, and rare diseases, reflecting greater therapeutic diversity beyond oncology dominance. Key innovation trends include first- and best-in-class biol...

Dec 23, 2025

Novo Nordisk Submits CagriSema for FDA Review as a Once-weekly GLP-1–amylin Combination Therapy for Weight Management Novo Nordisk has officially filed a New Drug Application (NDA) with the U.S. FDA for CagriSema, a once-weekly subcutaneous injection intended for chronic weight management. CagriSema represents a...

Aug 22, 2025

Over the past four decades, hemophilia A treatment has advanced remarkably. Beginning with the introduction of cryoprecipitate in 1964, treatment progressed to plasma-derived FVIII, then to recombinant FVIII therapies in the 1980s. The 1990s and 2000s saw the rise of Standard Half-life (SHL) products, followed by E...

Apr 07, 2025

Over the past three years, the FDA has approved six new hemophilia treatments, including three gene therapies. Now, Sanofi’s Qfitlia (fitusiran) enters the competitive hemophilia therapeutics market, distinguishing itself as the only therapy approved for all types of hemophilia. Unlike most existing treatments, Qfi...

Apr 01, 2025

Sanofi’s Qfitlia Becomes First US-Approved Therapy for Hemophilia A or B The FDA has approved Qfitlia (fitusiran), the first antithrombin-lowering therapy for routine prophylaxis to prevent or reduce bleeding episodes in hemophilia A or hemophilia B patients (ages 12+) with or without inhibitors. Approval is bas...

Oct 23, 2024

Plasma protein therapeutics are revolutionizing the landscape of modern medicine by harnessing the healing potential of the human body. Derived from donated blood plasma, these life-saving treatments are packed with antibodies, clotting factors, and other essential proteins that help manage a wide array of conditio...

Jul 14, 2023

Hemophilia A is a rare genetic bleeding disorder that primarily affects males. Hemophilia A is the most common type of hemophilia, accounting for approximately 80% of all cases. It is estimated to occur in about 1 in 5,000 to 10,000 male births. As per the Centers for Disease Control (CDC) (2020), the estimated pre...

Jul 04, 2023

FDA Approves First Gene Therapy for Severe Hemophilia A BioMarin Pharmaceutical Inc., a global biotechnology company dedicated to transforming lives through genetic discovery, announced that the US Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the tr...

Jun 09, 2023

The cell and gene therapy market has seen a revolutionary transition in recent years, with advancements in scientific research and novel methods of treatment driving a rise in development activities. This has resulted in an increase in the number of cell and gene therapy choices available to patients suffering from...

Nov 07, 2022

Hemophilia A treatment scenario before the launch of HEMLIBRA Despite the ups and downs throughout history, the management of hemophilia A patients substantially improved over the past 40 years. The groundbreaking discovery of cryoprecipitate in 1964 marked the beginning of the modern progression of hemophilia t...