Drugmakers' hopes for gene therapy rise despite tiny sales in Europe - (Nasdaq via NewsPoints Desk)

  • Drugmakers have increased hopes for gene therapies despite meagre sales in the two months after their launch in Europe, as reported NASDAQ Tuesday.

  • Among the treatments, UniQure's rare blood disorder therapy Glybera is being pulled from the market due to a lack of demand, while the future of GlaxoSmithKline's adenosine deaminase deficiency-severe combined immunodeficiency treatment Strimvelis is unclear after the company announced plans to review and divest its rare diseases unit.

  • "It's disappointing that so few patients have received gene therapy in Europe," said KPMG chief medical adviser Hilary Thomas, adding "it shows the business challenges and the problems faced by publicly-funded healthcare systems in dealing with a very expensive one-off treatment."

  • Meanwhile, Spark Therapeutics expects to receive US clearance next year for a gene therapy for treating a rare form of blindness, while Novartis could gain US approval as soon as September for its gene-modified cell therapy against leukaemia.

  • Although Spark CEO Jeffrey Marrazzo explained that the company hopes to avoid the mistakes of GlaxoSmithKline and UniQure in launching its gene therapy, new business models may be needed for gene therapy manufacturers, which also include BioMarin, bluebird bio and GenSight Biologics.

  • "Is it something that I think will take market share mid- to long-term if the data continues to be encouraging," questioned Shire CEO Flemming Ornskov, adding "yes. But I think everybody will have to figure out a business model."

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