![]() bluebird then broke its own pricing record when the $3m Skysona was approved, and now faces the unique challenge of selling the world’s two most expensive drugs. bluebird bio doubled the number of gene therapies available for inherited diseases within a one-month time span and snatched the title of owning the most expensive drug away from Novartis with Zynteglo’s price of $2.8m. The second gene therapy reached the market in 2019 Novartis’ Zolgensma was approved for spinal muscular atrophy (SMA) at a hefty price of $2.1m, making it the most expensive drug in the world. Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird’s approvals, there were only two FDA-approved gene therapies for inherited conditions on the market. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases. Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. Shortly after this, the FDA announced the accelerated approval of bluebird’s Skysona (elivaldogene autotemcel), or eli-cel, on 19 September. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.
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