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(Gene therapy for blindness) Drug could cost US$450,000

World | Oct 13, 2017 20:52
Philadelphia-based Spark Therapeutics hopes to use its technology to treat other retinal disorders, including one called choroideremia, which affects about three times as many people as the condition currently under Food and Drug Administration review.
The company is also investigating therapies to treat the blood disorder hemophilia and diseases of the nervous system.
As a potential breakthrough therapy for a rare disease, much of the discussion around Luxturna has focused on its cost. Drugmakers often price similar drugs at US$250,000 or more. Spark has not given an estimated cost, which companies usually announce only after approval. But the company’s stock ticker symbol "ONCE” reinforces the company’s hope that this will be a one-time treatment — and expense.
"It is the aspiration,” Spark Therapeutics CEO Jeff Marrazzo told The Associated Press prior to the meeting with the experts.
Pharmaceutical analyst Michael Yee estimates the drug will be priced between US$350,000 and US$450,000 per injection. He expects sales of US$40 million to US$50 million in 2018, growing to as much as US$100 million in 2019.-AP


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