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GlaxoSmithKline says it has treated a child with Strimvelis, its gene therapy for immune deficiency.
A child in Europe has become the second individual ever to receive a commercial gene therapy, according to GlaxoSmithKline.
The treatment, called Strimvelis, can provide an outright cure for a rare inherited immune deficiency by revising a patient's genetic makeup (see “Gene Therapy’s First Out-and-Out Cure Is Here”).
Gene therapy has been widely explored in experimental medical studies, but its commercial potential is largely untested. Prior to now, only a single other individual, also from Europe, had ever accessed gene therapy to treat an inherited ailment outside of a clinical trial. That individual received a different drug, Glybera, in 2015.
On Tuesday, GlaxoSmithKline spokesperson Anna Padula said the company treated its first patient in March, nearly a year after Strimvelis was approved for sale in Europe in May 2016. The company declined to provide the nationality of the patient or say how the treatment was paid for.
In March, GlaxoSmithKline’s project leader for Strimvelis, Jonathan Appleby, said difficulty organizing cross-border European reimbursement for Strimvelis...