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Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease
CAMBRIDGE, Mass., June 03, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., June 03, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). “Sickle cell disease is a devasting disorder that affects approximately 100,000 people in the U.S., leading to anemia, severe pain, stroke and even early death. Receiving orphan drug designation from the FDA emphasizes the importance of new treatment options for this debilitating disease, and our clinical data suggest that BEAM-101, the lead program in our hematology franchise, has the potential to offer a differentiated, best-in-class treatment,” said Amy Simon, M.D., chief medical officer of Beam. “We look forward to continuing to progress our BEACON Phase 1/2 clinical trial of BEAM-101 in patients with severe sickle cell disease and to working closely with the FDA with the goal of bringing BEAM-101 to patients as safely and quickly as possible, embodying our mission of providing lifelong cures to patients suffering from severe diseases.” The FDA’s orphan drug designation is designed to support the development and evaluation of treatments for rare diseases affecting fewer than 200,000 people in the U.S. The designation comes with potential benefits for the sponsor company, including tax credits for qualified clinical trials, exemption from user fees, and a potential seven years of market exclusivity after approval. Data from seven patients treated with BEAM-101 in the BEACON clinical trial were presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2024. Updated clinical data have been accepted for presentation at the European Hematology Association (EHA) 2025 Congress in June. Treatment with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis. The initial safety profile of BEAM-101 was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation. Manufactured in Beam’s North Carolina facility, ...