Pharma Focus America

FDA Grants Orphan Drug Status to BE-101, a Novel Engineered B Cell Therapy, for Hemophilia B Treatment

Wednesday, June 05, 2024

Be Biopharma, Inc. ("Be Bio") has achieved a significant milestone in advancing treatment options for Hemophilia B. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to BE-101, an innovative engineered B Cell Medicine (BCM) developed by Be Bio. This designation acknowledges the therapy's potential to address a rare condition affecting fewer than 200,000 individuals in the United States.

BE-101 represents a novel approach to treating Hemophilia B by introducing the human FIX gene into primary human B cells. This innovative technique allows for sustained expression of active FIX, offering a promising alternative to current treatment regimens. With the flexibility for re-dosing and titration, BE-101 aims to alleviate the infusion burden associated with existing therapies while potentially reducing bleeding rates and FIX usage.

Be Bio, emphasized the significance of BE-101's potential, stating, "Despite recent advancements, patients with hemophilia B continue to face challenges such as bleeding events and chronic pain. BE-101 has the potential to revolutionize treatment by providing durable and flexible therapy, marking a significant step forward in improving the lives of individuals with this condition."

Orphan Drug Designation offers several advantages, including exclusive marketing rights, exemption from user fees, and eligibility for tax credits on clinical trials. Be Bio is set to commence its Phase 1/2 study, BeCoMe-9, evaluating BE-101 in adults with severe or moderately severe Hemophilia B in the latter half of 2024.

Hemophilia B is a rare X-linked recessive bleeding disorder caused by mutations in the FIX gene. Current treatment options often require frequent infusions of exogenous FIX to maintain therapeutic levels. While gene therapy has emerged as a potential alternative, the need for improved therapeutic options remains critical.

BE-101 represents a pioneering approach in the field of cellular medicine, harnessing the power of engineered B cells to produce therapeutic proteins. This new class of Engineered B Cell Medicines (BCMs) holds promise across various therapeutic areas, offering the potential for durable, redosable, and personalized treatments. Be Bio's commitment to innovation underscores its dedication to transforming the landscape of biopharmaceuticals and improving patient outcomes.



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