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UK cost watchdog recommends Novartis' blindness therapy Luxturna

[September 04, 2019] (Reuters) - Novartis AG's gene therapy for blindness, Luxturna, is recommended for use on England's public health service, the country's healthcare cost-effectiveness watchdog NICE said on Tuesday.

Novartis owns the rights to sell the one-time gene therapy developed by Spark Therapeutics outside the United States.

The decision by the National Institute for Health and Care Excellence (NICE) comes as a boost to the Swiss drugmaker, which had come under regulatory scrutiny for data discrepancy issues related to its $2 million gene therapy, Zolgensma.

Novartis has been expanding into gene and cell therapies for rare diseases and licensed Luxturna last year, for $105 million upfront, up to another $65 million in milestone payments, and royalties on sales.

The gene therapy, injected directly into the retina, won EU approval last November, to treat patients suffering from inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder that causes vision loss and usually leads to blindness.

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In England, Luxturna carries a price tag of 613,410 pounds ($753,083.46) per patient, but the treatment is available to England's National Health Service at a confidential discount, NICE said.

The treatment was labeled too expensive in the United States by non-profit Institute for Clinical and Economic Review (ICER), which last year said it should cost about 50% to 75% less than its list price. (https://reut.rs/2ks7XCh)

The drug, which works by introducing a healthy copy of the defective RPE65 gene into the retinal cells, is priced at $850,000, or $425,000 per eye in the United States, making it one of the costliest drugs.

(Reporting by Manojna Maddipatla in Bengaluru; Editing by Shailesh Kuber)

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