Spark's Gene Therapy Is On The Cusp Of Approval; Now It Gets Interesting
Spark's Luxturna is all but guaranteed to reach the US market as the first gene therapy following a unanimous positive FDA advisory committee review. Discussions about cost and value are sure to intensify.
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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.
The pharmaceutical industry's manufacturing capacity for cell and gene therapies is under pressure from an ever-increasing number of life sciences companies wanting to exploit rare expertise in the sector, but CMOs and government-backed facilities are rapidly coming on-stream, hoping to develop local clusters of companies to deliver the complete "living medicines" supply chain.
Luxturna, a one-time treatment for a rare inherited form of blindness, would not be cost-effective at the $1m price level that has been suggested as a possibility, based on ICER's thresholds. But the small patient population would keep it from triggering ICER's US budget spending threshold.