Luxturna: the first licensed gene therapy for rare inherited eye disorder is recommended by NICE

eye disorder

Luxturna: the first licensed gene therapy for rare inherited eye disorder is recommended by NICE

A draft NICE guidance recommends Voretigene Neparvovec (also called Luxturna) to treat people with vision loss caused by inherited retinal dystrophy from confirmed RPE65 gene mutations who have enough viable retinal cells.

Voretigene Neparvovec is the first licensed gene therapy for vision loss caused by RPE65-mediated inherited retinal dystrophy and its evaluation at NICE has taken 20 weeks instead of the average 38 weeks within the Highly Specialised Technologies programme.

Around 86 people would be eligible for treatment in England, where the product’s price breached a budget impact test. However, a commercial arrangement between Novartis and NHS England made the treatment available to the NHS. Without this deal, the therapy would have breached the regulator’s budget impact test, because the product’ list price is £613,410 per patient.

This is the second innovative gene therapy which NICE has produced positive guidance on in its Highly Specialised Technologies programme. – after Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency was recommended in February last year.

The draft guidance is now out for consideration by the company, healthcare professionals and patient groups. If there are no appeals, NICE expects to publish final guidance next month.

More information: 

Are you working in gene therapy and would like additional regulatory support? Please contact Asphalion at [email protected]

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