Betibeglogene autotemcel, sold under the brand name Zynteglo, is a gene therapy for the treatment for beta thalassemia.[1][5][2] It was developed by Bluebird Bio and was given breakthrough therapy designation by the US Food and Drug Administration in February 2015.[6][7]
The most common adverse reactions include reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nosebleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus (itch).[5]
It was approved for medical use in the European Union in May 2019,[2] and in the United States in August 2022.[5]
^ abc"Zynteglo dispersion for infusion - Summary of Product Characteristics (SmPC)". (emc). 12 May 2020. Retrieved 3 January 2021.[permanent dead link]
^ ab"Zynteglo- betibeglogene autotemcel suspension". DailyMed. 26 August 2022. Retrieved 19 November 2022.
^"Zynteglo". U.S. Food and Drug Administration. 17 August 2022. Archived from the original on 26 August 2022. Retrieved 26 August 2022.
^ abcd"FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions". U.S. Food and Drug Administration (FDA) (Press release). 17 August 2022. Archived from the original on 21 August 2022. Retrieved 20 August 2022. This article incorporates text from this source, which is in the public domain.
^"Ten things you might have missed Monday from the world of business". The Boston Globe. 3 February 2015. Archived from the original on 1 August 2020. Retrieved 13 February 2015.
^"Lentiviral vectors". 27 June 2019. Archived from the original on 21 August 2022. Retrieved 8 July 2019.
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