Gene Therapy: 5 Drugs in the Top Ten, Most Expensive at $3.5 Million

In the current global pharmaceutical market, gene therapy drugs occupy a significant portion of the most expensive drug rankings, with five listed in the top ten. Pfizer's recently approved treatment for moderate to severe Hemophilia B, Beqvez, is priced at a staggering $3.5 million, making it one of the highest-priced drugs in history.

Hemophilia is a hereditary bleeding disorder primarily divided into Hemophilia A and B, caused by mutations in the genes for clotting factors VIII and IX, respectively. Patients typically suffer from lifelong coagulation dysfunction, leading to joint bleeding and other severe complications. Currently, replacement therapies with clotting factors are the main treatment method, but frequent long-term injections can lead to poor patient compliance and may produce inhibitory antibodies, affecting treatment efficacy. Although new clotting factor drugs, humanized bispecific antibodies, and monoclonal antibodies that inhibit the tissue factor pathway have made advancements in administration methods and duration of action, these methods still cannot cure hemophilia.

For hemophilia patients, gene therapy offers a glimmer of hope for a cure. Through gene therapy, clotting factors can be safely delivered into the patient's cells for long-term stable expression, thereby improving bleeding conditions and coagulation function, which is the ultimate goal of hemophilia treatment.

Currently, several gene therapy drugs for hemophilia have been approved, including:

• HYMPAVZI™ (Matizumab): An anti-TFPI antibody suitable for patients with Hemophilia A or B, developed by Pfizer, approved by the European Commission in November 2024.
• Vonvendi (Veyinzh®): A recombinant von Willebrand factor (rVWF) for adult patients with von Willebrand disease (VWD), developed by Takeda, approved by China's National Medical Products Administration in August 2024.
• Beqvez: A neutralizing antibody for adult patients with moderate to severe Hemophilia B, developed by Pfizer, priced at $3.5 million.
• Hemgenix (AMT060): A gene therapy based on adeno-associated virus 5 (AAV5) for patients with Hemophilia B, developed by UniQure, approved by the U.S. FDA in November 2022.
• Roctavian: Also based on the AAV5 vector, this gene therapy is for patients with Hemophilia A (HA), priced at $2.9 million per treatment, developed by BioMarin, and received conditional marketing authorization from the European Commission in August 2022.

As the hemophilia market continues to grow, it’s expected to reach a scale of billions by 2028. Currently, over 40 gene therapies for hemophilia are in research, with multinational companies like Pfizer, Sanofi, Bayer, and Takeda, as well as domestic firms like China National Pharmaceutical Group and Hualan Biologicals, actively conducting clinical studies. Despite the enormous potential of gene therapy in treating hemophilia, the high costs remain a significant barrier to its widespread adoption.