Zynteglo (betibeglogene autotemcel) · bluebird bio

ZYNTEGLO is an autologous gene therapy which includes hematopoietic stem cells (HSCs) that have been genetically modified ex vivo . The nature of ZYNTEGLO is such that conventional studies on pharmacokinetics, absorption, distribution, metabolism, and elimination are not applicable.

The efficacy of ZYNTEGLO was evaluated in 2 ongoing Phase 3 open-label, single-arm, 24-month, multicenter studies (Study 1 and Study 2) in 41 patients aged 4 to 34 years with β-thalassemia requiring regular transfusions. Following completion of the 24-month parent studies, patients were invited to enroll in an ongoing long-term safety and efficacy follow-up study for an additional 13 years (Study 3). Patients were considered to be eligible for the Phase 3 studies if they had a history of transfusions of at least 100 mL/kg/year of packed red blood cells (pRBCs) or with 8 or more transfusions of...

The following adverse reactions are described elsewhere in the labeling: Delayed Platelet Engraftment [see Warnings and Precautions (5.1) ] Risk of Neutrophil Engraftment Failure [see Warnings and Precautions (5.2) ] Risk of Insertional Oncogenesis [see Warnings and Precautions (5.3) ] Hypersensitivity Reactions [see Warnings and Precautions (5.4) ] The most common non-laboratory adverse reactions (incidence ≥ 20%) were mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair lo...

None. None. ( 4 )

Delayed Platelet Engraftment: Monitor platelet counts until platelet engraftment and recovery are achieved. Patients should be monitored for thrombocytopenia and bleeding. ( 5.1 ) Risk of Neutrophil Engraftment Failure: Monitor absolute neutrophil counts (ANC) after ZYNTEGLO infusion. If neutrophil engraftment does not occur administer rescue cells. ( 5.2 ) Risk of Insertional Oncogenesis: Monitor patients at least annually for hematologic malignancies for at least 15 years after ZYNTEGLO infusi...

7 DRUG INTERACTIONS No formal drug interaction studies have been performed. ZYNTEGLO is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters. Anti-retrovirals and Hydroxyurea : Do not take anti-retroviral medications or hydroxyurea for one month prior to mobilization, or for the expected duration for elimination of the medications, and until all cycles ...