The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Be Biopharma’s (Be Bio) BE-101, a novel engineered B Cell medicine (BCM) being developed for the treatment of hemophilia B. Be Bio is on track to initiate its Phase 1/2 study, BeCoMe-9, evaluating BE-101 in adults with severe or moderately severe hemophilia B in the second half of 2024.
“Despite recent advances, patients with hemophilia B still suffer from bleeding events, joint damage and chronic pain,” said Joanne Smith-Farrell, Ph.D., Chief Executive Officer of Be Bio. “BE-101 has the potential to be the first and only Factor IX replacement therapy that is extremely durable, re-dosable and titratable, representing an opportunity to dramatically improve the treatment paradigm in hemophilia B. The Orphan Drug Designation reinforces the potential of this therapy and underscores the need for improved therapeutic options for these patients.”
The FDA grants Orphan Drug Designation to drugs or biologics designed to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. This designation offers several significant advantages, including a seven-year period of exclusive marketing rights following approval, exemption from user fees, and eligibility for tax credits on qualified clinical trials.
