Transfusion-dependent beta-thalassemia gene therapy approved in Europe - Healing Genes

Transfusion-dependent beta-thalassemia gene therapy approved in Europe

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Zynteglo is Bluebird’s first drug to be approved and is the first gene therapy to be cleared for use in transfusion-dependent beta-thalassemia.

Its marketing authorization from the European Commission follows landmark approvals from the Food and Drug Administration for Novartis’ Zolgensma and Spark Therapeutics’ Luxturna, which respectively treat spinal muscular atrophy and a type of inherited eye disease.

Taken together, the therapies illustrate the rapid advances made in gene therapy over the past several years, as more and more diseases appear treatable by correcting dysfunctional genes. Commercializing such therapies is another question, though, given high costs of treatment and complex production.

The conditions all three drugs treat are rare, with only a few hundred to a few thousand patients diagnosed each year. In Bluebird’s case, estimates put the number of people in the EU who could initially receive Zynteglo at between 2,000 and 3,000.

The European Commission cleared Zynteglo for a specific genotype of transfusion-dependent beta-thalassemia, and for those patients aged 12 and older who are able to receive a stem cell transplant but have no matched donor.

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