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Regulating therapies for rare diseases – recent approvals
This article summarises some of the recent notable drug approvals in the EU and US, including a gene therapy and an enzyme replacement therapy.
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Beta thalassaemia
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FDA approves Zynteglo gene therapy for beta-thalassemia patients
bluebird bio’s Zynteglo (betibeglogene autotemcel), a one-time treatment for transfusion-dependent beta-thalassemia, will roll out with a $2.8mn price tag per dose.
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β-thalassemia patients achieve transfusion independence with gene therapy
Over 90 percent of β-thalassemia patients treated with Betibeglogene autotemcel (beti-cel) achieved transfusion independence lasting over a year.
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beti-cel gene therapy demonstrates durable efficacy in beta thalassaemia patients
The betibeglogene autotemcel (beti-cel) gene therapy caused paediatric and adult beta thalassaemia patients to achieve transfusion independence lasting over two years.
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Marketing of Zynteglo suspended as precaution due to potential cancer risk
bluebird bio has chosen to suspend sales of Zynteglo while they investigate whether safety concerns identified with a related investigational gene therapy may apply to the licenced medicine.
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EMA recommends eight drugs for approval from April meeting
The EMA's human medicines committee has adopted a positive opinion for eight medicines, recommending their marketing authorisation.
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EMA announces medicine approval recommendations from November meeting
The EMA CHMP has revealed its suggestion to grant marketing authorisation to seven medicines after its most recent meeting.
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Reblozyl approved for beta thalassaemia treatment
The US Food and Drug Administration (FDA) has granted Orphan Drug designation to Reblozyl for the treatment of anaemia in adults with beta thalassaemia.
