NICE backing Novartis’s gene therapy Luxturna

Luxturna, a gene therapy used to treat a rare, inherited form of blindness will now be available for routine use in the NHS in England and Wales.

It is a one-off treatment and it was backed last November for both children and adults who are living with vision loss due to genetic mutations. It is estimated to affect around 180 patients and patients almost invariably end up losing their sight entirely.

Along with the gene therapy, patients will have to have sufficient retinal cells remaining for the treatment, which will involve a one-time injection under the retina. The company and NHS have agreed on a confidential discount of the list price of £613,410 for the therapy.

Novartis secured the rights for the treatment outside of US from its original developer Spark Therapeutics last year, shortly after the biotech received FDA approval for Luxturna. Since this approval. Roche has been hot on Spark Therapeutics heels in trying to take over the biotech firm, in a deal estimated at $4.3bn. However the deal has been setback multiple times due to anti-trust concerns by regulators from the UK and US.

The Scottish Medicines Consortium will deliver its verdict on the gene therapy in December.

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