Spark's Luxturna Approval Ushers In A New Gene Therapy Era
Spark's gene therapy for patients with an inherited form of blindness will be available in the US late in the first quarter, but on the big question – whether it will cost $1m for the one-time treatment – Spark would not say.
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One regenerative medicine for an ultra-rare disease may have a small impact, but payers will be flooded with dozens of new cell and gene therapies over the next few years. Enzyvant, Novartis and Spark share how they are positioning their complex products.
The first of what promises to be a wave of potentially curative gene therapies are coming to market. One of the challenges for sponsors will be how to deliver long-term outcomes data demanded by regulators and payors from patients who don’t have any reason to go to the doctor.
It has become standard practice for manufacturing to get early attention in the cell and gene therapy development, but logistics is often not considered until closer to commercialization, which could be a strategic mistake given the speed with which new treatments are being approved.