#China #Shanghai #Belief BioMed #Takeda China #BBM-H901

Introduction

In a groundbreaking development for hemophilia B treatment in China, Belief BioMed and Takeda China jointly announced the official approval of BBM-H901 (Dalnacogene Ponparvovec Injection) by the National Medical Products Administration (NMPA). This approval marks a significant milestone as it is the first gene therapy specifically designed for this condition in the country.

Understanding Hemophilia B

Hemophilia B, an inherited bleeding disorder resulting from a deficiency of coagulation factor IX (FIX), often leads to excessive bleeding and complicated joint damage. For years, patients have depended on traditional methods like prothrombin complex concentrate (PCC) or FIX replacement therapies, resulting in frequent treatments and substantial economic burdens on families. With the introduction of BBM-H901, sufferers of this condition are offered a revolutionary approach.

The Innovative Approach of BBM-H901

BBM-H901 utilizes a recombinant adeno-associated virus (rAAV) vector to effectively deliver an optimized gene that encodes human FIX directly to the liver cells of patients. This allows for continuous expression and secretion of FIX, significantly promoting blood coagulation and reducing the risk of joint damage and bleeding incidents.

Dr. Xiao Xiao, Co-founder and Chief Science Officer of Belief BioMed, expressed the company’s commitment to addressing unmet medical needs through innovative research and development. He emphasized that BBM-H901 represents a new treatment avenue that has the potential to restore a normal life for hemophilia B sufferers, indicating the company’s dedication to accelerating both local and international commercialization processes for this therapy.

Clinical Studies and Results

The efficacy of BBM-H901 has been documented through extensive clinical trials. The first Investigator-initiated Trial (IIT) commenced in 2019, with findings published in reputable journals including The Lancet-Hematology and The New England Journal of Medicine. Data from trials showed promising results, with patients achieving a mean FIX activity of 36.9 IU/dL after treatment, and no serious adverse events were reported.

More recent follow-ups demonstrated stable FIX activity and a dramatic reduction in the rate of bleeding episodes, falling from an average of 58.2 times per year to just 2.9 after gene therapy treatment. Dr. Zhang Lei, a leading investigator in the clinical studies, highlighted the therapy as a revolutionary option, stating that it not only simplifies the treatment regimen but also carries the potential for improved quality of life.

Future Steps and Expectations

Both Belief BioMed and Takeda China plan to collaborate on accelerating the commercialization of BBM-H901 across mainland China, Hong Kong, and Macau. Takeda’s Senior Vice President Sean Shan mentioned the goal of enriching the therapeutic landscape for hemophilia and rare diseases, reinforcing their strategy to expedite the introduction of groundbreaking therapies to those in need.

Furthermore, both companies aspire towards expanding the global reach of this innovative therapy, hoping to assist more hemophilia B patients worldwide. With the introduction of BBM-H901, the future of hemophilia treatment is set for a positive transformation in China and beyond.