Cornering The Market On Innovation? The First CAR-T And The First Gene Therapy
Executive Summary
It isn’t just the cutting-edge science. Novartis’ landmark approval also highlights regulatory and reimbursement innovation and also may set the rules that the next wave of cell and gene therapies have to follow.
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Three of the accelerated approvals were for CAR-T products, a class that the agency considers to be gene therapies. The FDA’s September approval of bluebird bio’s Skysona for cerebral adrenoleukodystrophy was the lone non-cancer product in the group. Pink Sheet infographic tracks the required confirmatory studies and timelines for all four products.
A New Drug Price Milestone
Much like Genzyme’s Ceredase did two decades ago, Novartis’ Zolgensma is setting a new pricing benchmark. The first million-dollar product is launching in the US and it will actually cost more than $2 million.
Spark Avoids Igniting Pricing Firestorm With Savvy Launch Messages
US launch of gene therapy Luxturna could set tone for attention to extremely high priced therapies in 2018. So far, company seems to be doing things right.