United States - English - NLM (National Library of Medicine)

bluebird bio, inc. - betibeglogene autotemcel (unii: mee8487rtp) (betibeglogene autotemcel - unii:mee8487rtp) - zynteglo is indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (rbc) transfusions. none. risk summary there are no available data with zynteglo administration in pregnant women. consider the risks associated with myeloablative conditioning agents on pregnancy and fertility. no reproductive and developmental toxicity studies in animals have been conducted with zynteglo to assess whether it can cause fetal harm when administered to a pregnant woman. it is not known whether zynteglo has the potential to be transferred to the fetus. therefore, zynteglo should not be administered to women who are pregnant, and pregnancy after zynteglo infusion should be discussed with the treating physician. no nonclinical germline transmission studies have been conducted with zynteglo. in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. risk summary there is no information regarding the presence of zynteglo in human milk, the effect on the breastfed infant, and the effects on milk production. the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for zynteglo and any potential adverse effects on the breastfed child from zynteglo. therefore, zynteglo is not recommended for women who are breastfeeding, and breastfeeding after zynteglo infusion should be discussed with the treating physician. pregnancy testing a negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before zynteglo administration. contraception there are insufficient exposure data to provide a precise recommendation on duration of contraception following treatment with zynteglo. women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra-uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of zynteglo. advise patients of the risks associated with conditioning agents. infertility there are no data on the effects of zynteglo on fertility. data are available on the risk of infertility with myeloablative conditioning. advise patients of the option to cryopreserve semen or ova before treatment, if appropriate. the safety and efficacy of zynteglo have been established in pediatric patients with β-thalassemia requiring regular transfusions. use of zynteglo is supported by two phase 3 studies [see clinical studies (14)] that included 27 pediatric patients in the following age groups: 16 children (less than 12 years) and 11 adolescents (age 12 years to less than 18 years). no differences in efficacy or clinical safety were observed between the adult and pediatric subgroups. engraftment times were longer in pediatric patients, but not associated with increases in infections or bleeding events. the median (min, max) time to neutrophil engraftment for patients less than 18 years was 26 (16, 39) days versus 21 (13, 27) days for patients 18 years or older. the median (min, max) time to platelet engraftment for patients less than 18 years was 50 (20, 94) days versus 43 (21, 58) days for patients 18 years or older. longer engraftment time was associated with intact spleens. the safety and efficacy of zynteglo in children less than 4 years of age have not been established. no data are available. zynteglo has not been studied in patients > 65 years of age. hematopoietic stem cell (hsc) transplantation must be appropriate for a patient to be treated with zynteglo. zynteglo has not been studied in patients with hiv-1, hiv-2, htlv-1, or htlv-2. a negative serology test for hiv is necessary to ensure acceptance of apheresis material for zynteglo manufacturing. apheresis material from patients with a positive test for hiv will not be accepted for zynteglo manufacturing. zynteglo has not been studied in patients with renal impairment. patients should be assessed for renal impairment, defined as creatinine clearance ≤ 70 ml/min/1.73 m2 , to ensure hsc transplantation is appropriate. zynteglo has not been studied in patients with hepatic impairment. patients should be assessed for hepatic impairment to ensure hsc transplantation is appropriate.

European Union - English - EMA (European Medicines Agency)

bluebird bio (netherlands) b.v. - autologous cd34+ cell enriched population that contains hematopoietic stem cells transduced with lentiglobin bb305 lentiviral vector encoding the beta-a-t87q-globin gene - beta-thalassemia - other hematological agents - zynteglo is indicated for the treatment of patients 12 years and older with transfusion-dependent β thalassaemia (tdt) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (hsc) transplantation is appropriate but a human leukocyte antigen (hla)-matched related hsc donor is not available.

United States - English - NLM (National Library of Medicine)

bluebird bio, inc. - lovotibeglogene autotemcel (unii: 2c6a9nh2z8) (lovotibeglogene autotemcel - unii:2c6a9nh2z8) - lyfgenia is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. limitations of use following treatment with lyfgenia, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions [see adverse reactions (6.1)] . lyfgenia has not been studied in patients with more than two α-globin gene deletions. none. risk summary there are no available data on lyfgenia administration in pregnant women. consider the risks associated with myeloablative conditioning agents on pregnancy and fertility. no reproductive and developmental toxicity studies in animals have been conducted with lyfgenia to assess whether it can cause fetal harm when administered to a pregnant woman. it is not known whether lyfgenia has the potential to be transferred to the fetus. therefore, lyfgenia should not be administered to women who are pregnant, and pregnancy after lyfgenia infusion should be discussed with the treating physician. in the u.s. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2%-4% and 15%-20%, respectively. risk summary there is no information regarding the presence of lyfgenia in human milk, the effect on the breastfed infant, and the effects on milk production. the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for lyfgenia and any potential adverse effects on the breastfed child from lyfgenia. therefore, lyfgenia is not recommended for women who are breastfeeding, and breastfeeding after lyfgenia infusion should be discussed with the treating physician. pregnancy testing a negative serum pregnancy test must be confirmed prior to the start of mobilization and re‑confirmed prior to conditioning procedures and before lyfgenia administration. contraception there are insufficient exposure data to provide a precise recommendation on duration of contraception following treatment with lyfgenia. women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra-uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of lyfgenia. advise patients of the risks associated with conditioning agents. infertility there are no data on the effects of lyfgenia on fertility. data are available on the risk of infertility with myeloablative conditioning. advise patients of the risks and the options for fertility preservation. the safety and efficacy of lyfgenia have been established in pediatric patients 12 years of age and older with sickle cell disease, including 8 adolescents (age 12 years to less than 18) [see clinical studies (14)]. no clinically meaningful differences in efficacy or safety were observed between the adult and pediatric subgroups. the safety and efficacy of lyfgenia in children less than 12 years of age have not been established. no data are available. lyfgenia has not been studied in patients 65 years of age and older. autologous hematopoietic stem cell (hsc) transplantation must be appropriate for a patient to be treated with lyfgenia. lyfgenia has not been studied in patients with hiv-1 or hiv-2. a negative serology test for hiv is necessary prior to apheresis. patients with a positive test for hiv will not be accepted for lyfgenia treatment. lyfgenia has not been studied in patients with renal impairment (defined as creatinine clearance ≤ 70 ml/min/1.73 m2 ). patients' renal function should be assessed for renal impairment to ensure autologous hsc transplantation is appropriate. lyfgenia has not been studied in patients with advanced hepatic disease. patients' hepatic function should be assessed for hepatic impairment to ensure autologous hsc transplantation is appropriate.