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The FDA approves the betibeglogene autotemcel for pediatric and adult patients β-thalassemia and requiring regular infusions
According to the FDA press release, the FDA has approved betibeglogene autotemcel (beti-cel; Zynteglo) is used in adults and children who require regular infusions of red blood cells β-thalassemia[1
Patients will receive beti-cel as a 1x gene therapy
"Beti-cel is a gene therapy
Approval is supported
HGB-207/Northstar-2
In the phase 3 HGB-207/Northstar-2 trial (NCT02906202), 20 of the 23 adult or pediatric patients with transfusion-dependent β-thalassemia and non-beta 0/β0 genotype achieved transfusion independence (TI), with 6 patients having an average haemoglobin level of 11.
Four patients experienced treatment-related adverse events
LTF-303 study
After the Stage 1/2 HGB-205 (NCT02151526) or Phase 3 HGB-207 trial, patients are invited to participate in a 13-year long-term LTF-303 study (NCT02633943)[4
Transfusion independence was achieved in 40 of the 51 patients, with 68% of all patients treated in the phase 1/2 trial and 86%
Pediatric research
The Phase 3 Northstar-2 and Northstar-3 (NCT03207009) study included pediatric patients, with 16 under 12 years of age and 11 between the ages of 12 and
The incidence of treatment-related AE was low in both studies, with one patient developing grade 3 thrombocytopenia after transfusion
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