Sickle cell disease cell therapy named FDA orphan drug
Therapy is designed to turn on production of fetal hemoglobin in blood cells
BEAM-101, a genetically modified cell therapy for sickle cell disease (SCD) that’s being tested in clinical trials, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA).
The FDA gives the designation to experimental therapies designed to treat conditions that affect fewer than 200,000 people in the U.S. The designation seeks to provide extra economic incentives to companies developing treatments for rare diseases, which, by definition, have a small market. Among the perks of orphan drug status is a guarantee that, if BEAM-101 is approved, its developer Beam Therapeutics would be entitled to seven years of market exclusivity.
“Sickle cell disease is a devastating 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,” Amy Simon, MD, chief medical officer of Beam, said in a company press release.
SCD is caused by mutations in the gene that provides instructions to make a component of the adult version of hemoglobin, the protein that red blood cells use to carry oxygen through the body. The abnormal protein tends to clump up in red blood cells, deforming them and making them prone to getting trapped inside blood vessels, which can lead to painful vaso-occlusive crises (VOCs) and other SCD symptoms and complications.
How is BEAM-101 expected to work in sickle cell disease?
An alternative form of hemoglobin called fetal hemoglobin (HbF) is produced during early fetal development, but normally stops being made shortly after birth. BEAM-101 is designed to turn on the production of HbF in blood cells, which should counteract the effects of the mutated adult hemoglobin.
As part of the treatment, a patient’s hematopoietic stem cells, which are responsible for giving rise to new blood cells, are collected then lab modified to turn on HbF production before being transplanted back into the patient to give rise to new HbF-producing blood cells. Before the transplant, patients must be treated with a chemotherapy agent called busulfan to wipe out existing hematopoietic stem cells and make room for the modified cells.
Beam is sponsoring a Phase 1/2 clinical trial called BEACON (NCT05456880) that’s testing BEAM-101 in adults with SCD, ages 18-35, who’ve had at least four VOCs in the two years before screening.
Early data presented last year indicated BEAM-101 increased HbF production as designed. The treatment was generally tolerated well, with no serious safety problems related to the therapy, though one participant had a fatal reaction deemed likely related to busulfan.
“Our clinical data suggest that BEAM-101 … has the potential to offer a differentiated, best-in-class treatment,” Simon said. “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.”
Beam plans to present updated data from BEACON this month at the European Hematology Association (EHA) 2025 Congress. The company said it’s expecting to have dosed 30 participants by mid-year.
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