The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending approval of GlaxoSmithKline's Strimvelis, also known as GSK2696273, to treat patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). The company noted that the medicine is a stem cell gene therapy created for an individual patient from their own cells and "is intended to correct the root cause of the disease."
Martin Andrews, head of the rare diseases unit at GlaxoSmithKline, noted that if approved, Strimvelis would be "the first corrective ex-vivo gene therapy for children to achieve regulatory approval anywhere in the world." Meanwhile, R&D president Patrick Vallance said the company also hopes "to apply this gene therapy platform technology across other diseases."
GlaxoSmithKline cited data from a pivotal trial of 12 children demonstrating a survival rate of 100 percent at three years post-treatment with Strimvelis, with 92 percent achieving interventional-free survival. Moreover, all 18 Strimvelis-treated children who contributed data to the marketing authorisation application are alive today with a median follow-up duration of roughly seven years. The drugmaker noted that the first of these patients had received the gene therapy over 13 years ago, while interventional-free survival within the evaluable population of 17 patients was 82 percent.
The gene therapy is only administered once and does not rely on a third-party donor, so there is no risk of graft versus host disease, the UK drugmaker noted. Strimvelis was developed by Ospedale San Raffaele and Fondazione Telethon through their joint San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), and was advanced by GlaxoSmithKline via a strategic partnership forged in 2010.
Andrews indicated that the company would adopt a "flexible approach" with regard to pricing for the gene therapy, including the potential for annuity-style payments spread over time. "We may well have a menu of options," he said, adding "some of the payers say they would rather have a one-off, upfront payment and that's fine by us, [while] others want to look at different mechanisms." European regulators cleared uniQure Pharmaceuticals' gene therapy Glybera (alipogene tiparvovec) for lipoprotein lipase deficiency in 2012, and the product was launched there two years later at a price of 1.1 million euros ($1.2 million). However, while GlaxoSmithKline is not disclosing the price on its product, a source close to the company suggested that, if approved, it would cost "very significantly less than $1 million."
For related analysis, see ViewPoints: Glybera broke down some barriers for gene therapy, but plenty more to go.
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