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Novartis-Spark gene therapy for rare eye disease gets nod from UK price watchdog

Novartis AG and Spark Therapeutics Inc.'s gene therapy Luxturna was recommended by the U.K.'s National Institute for Health and Care Excellence to treat certain patients with a rare inherited eye disorder.

The drug pricing watchdog said in a draft guidance that Luxturna, or voretigene neparvovec, will be made available on the U.K.'s National Health Service as a treatment for vision loss caused by inherited retinal dystrophy from RPE65 gene mutations in patients who have enough working retinal cells.

The RPE65 gene directs production of a certain protein that is essential for normal vision. The disease is progressive and leads to total blindness in most patients. Luxturna, which is injected directly into a patient's retina, replaces the defective RPE65 gene and enables the production of vision-essential protein.

The drug is approved in the EU and U.S. for the vision disorder. In January 2018, Swiss drugmaker Novartis acquired ex-U.S. rights to Luxturna from Philadelphia-based Spark Therapeutics.

According to NICE, the exact number of people affected by the disease is uncertain, but Novartis' UK unit estimated that about 86 patients are eligible to receive treatment in England.

The one-time gene therapy is priced £613,410 per patient. NICE said at the original price the technology failed the budget impacting test, but Novartis agreed to a confidential discount to make the treatment available to NHS.

NICE evaluated Luxturna under the Highly Specialised Technologies program that reduced the time period to 20 weeks from the average 38 weeks.

The agency expects to publish final guidance in October if no appeals are filed by the consultees, which include the company and healthcare professionals, among others.

Roche Holding AG is in the process of acquiring Spark Therapeutics.