Confidence in gene therapy grows, as GlaxoSmithKline offer their latest treatment Strimvelis, a therapy for severe combined immunodeficiency, with a money-back guarantee
Pharmaceuticals are complicated to produce and test. Metabolic pathways are interactive, fluid constructs that change and adapt to any external influence. This makes targeting any disease with specific pharmaceuticals exceptionally challenging; the body often reacts in undesirable ways, and individual differences can confound studies. Gene therapy is different in many respects - at least when it comes to monogenic disease in which one mutated gene is known to cause a condition. The great thing about gene therapy is not only that it offers permanent cures for these deadly conditions, but that if it works it's a simpler, more elegant concept of treatment. Fix the gene and you've fixed the disease (in many cases anyway).
A cure or a refund
The first gene therapy drug approved in the West is Glybera, which came in at a whopping $1 million price tag and targets lipoprotein lipase deficiency. GSK's Strimvelis instead comes in at a considerable 594,000 euros ($665,000), remaining one of the most expensive single therapies in the world but having come down considerably.
“The drug has to deliver what you say or we don’t pay,” Luca Pani, director general of the Italian Medicines Agency, told the publication. “If it does not work, they will return the money”
David Vetter, who sadly died at the age of 12 in 1984, was known as the 'bubble boy', and was born with severe combined immunodeficiency.
Strimvelis will only be distributed in Milan, Italy, and was developed in collaboration with Italy’s San Raffaele Telethon Institute for Gene Therapy. Italy has a history of establishing medicine deals with refund clauses, collecting over 200 million euros from pharmaceutical companies in 2015 from ineffective medicines.
A step in the right direction
GSK has admitted that with around 8 patients per year expected to seek and afford treatment, it does not expect to make a profit on the sales, but that the developed technology will help create additional gene therapy products. Given that the patients will likely be almost exclusively from European nations with generous national health care provision, this is not a medicine for those around the world who need treatment, However, gene therapy remains extremely expensive to produce, so until this cost barrier comes down price tags will remain in the hundreds of thousands for the time being. However, the concept of a cure, or your money back is a unique and encouraging concept - indicating new confidence in the proficiency of gene therapy technology. The field has certainly come a long way since the tragic death of Jesse Gelsinger following adenoviral treatment in 1999.
Read more at FiercePharma
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