Avrobio gets orphan drug nod for Fabry disease gene therapy

By The Science Advisory Board staff writers

October 29, 2020 -- Avrobio has been granted orphan drug designation by the European Commission for AVR-RD-01, an ex vivo lentiviral gene therapy that consists of a patient's own hematopoietic stem cells that are genetically modified to express alpha-galactosidase A, the enzyme that is deficient in patients with Fabry disease.

The drug has also received orphan drug designation from the U.S. Food and Drug Administration and is being evaluated in a phase II clinical trial.

Fabry disease is a rare, inherited lysosomal storage disorder characterized by toxic accumulation of globotriaosylceramide in the body due to a variation in the GLA gene.

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