Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
Phase of Trial: Phase III
Latest Information Update: 08 Feb 2019
At a glance
- Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
- Indications Beta-thalassaemia; Haemoglobinopathies; Sickle cell anaemia
- Focus Adverse reactions; Pharmacodynamics; Therapeutic Use
- Sponsors bluebird bio
- 05 Oct 2018 Based on the study HGB-204, HGB-205, HGB-207 and long-term follow-up study LTF-303, the European Medicines Agency (EMA) accepted the company's marketing authorization application (MAA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta/beta genotype
- 06 Aug 2018 Planned number of patients changed from 86 to 94.
- 26 Jul 2018 According to a bluebird bio media release, based on the study HGB-204, HGB-205, HGB-207 and long-term follow-up study LTF-303, the Committee for Medicinal Products for Human Use (CHMP) of EMA has granted the Accelerated Assessment for its marketing authorization application (MAA) for LentiGlobin gene therapy for the treatment of adolescent and adult patients with transfusion-dependent beta-thalassemia (TDT). The company is intend to file an MAA in 2018.