Be Biopharma (Be Bio) announced that the FDA has granted Orphan Drug Designation to BE-101, an engineered B Cell Medicine (BCM) for Hemophilia B. The designation provides exclusive marketing rights, user fee exemption, and clinical trial tax credits.
BE-101 is designed to provide sustained therapeutic FIX activity with a single infusion, potentially eliminating the frequent infusions required by current Hemophilia B treatments. This could lead to reduced bleeding rates and lower usage of FIX replacement therapies.
Hemophilia B, an X-linked bleeding disorder affecting males, occurs due to mutations in the FIX gene. Despite advances, patients still experience bleeding, joint damage, and chronic pain.
Be Bio’s Phase 1/2 study, BeCoMe-9, evaluating BE-101 in patients with severe or moderately severe Hemophilia B, is expected to begin in the second half of 2024. The study aims to demonstrate the efficacy and safety of BE-101 as a long-lasting alternative to current treatments.
Engineered B Cell Medicines (BCM) leverage the B cell’s ability to continuously produce high levels of proteins. This approach can potentially revolutionize the treatment paradigm in various diseases, including Hemophilia B, by offering a single-infusion, durable therapy that can be re-dosed.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
