This past quarter saw two new gene therapies: Novartis AG subsidiary AveXis, Inc.’s Zolgensma (onasemnogene abeparvovec-xioi) received FDA approval May 24 for the treatment of spinal muscular atrophy (RSP 6/19, p. 1), and bluebird bio’s Zynteglo (autologous CD34+ cells encoding βA-T87Q- globin gene) received conditional marketing authorization from the European Commission for transfusion- dependent beta thalassemia. While only a handful of therapies in the broader regenerative medicine/advanced therapy (RM/AT) space are available globally, a new report shows that is likely to change, as there are more than 1,000 products in the pipeline. Many of these therapies truly are innovative products, but they don’t come cheap and often have complex reimbursement schemes that some payers and providers are struggling with, an issue that will need to be handled soon as more of these therapies are poised to hit the market.
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