In January of this year, Novartis bought the ex-US rights for Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl) for $105m (€92.7m), with a further $65m in milestone payments potentially due.
Spark received US Food and Drug Administration approval at the end of last year, with the treatment becoming the first gene therapy available to treat patients with a genetic disease. It has now received approval from the European Commission for treatment of the same condition.
Luxturna is a one-time therapy designed to restore functional vision in children and adult patients with biallelic mutations of the RPE65 gene.
Mark Toms, CSO of Novartis UK, described how the treatment functions: “[Luxturna] provides a functioning copy of the RPE65 gene to act in place of the mutated RPE65 gene. This functioning gene produces the RPE65 protein, which has the potential to prevent RPE65 patients’ progression to complete blindness by improving visual acuity.”
In the US, Spark prices the product at $425,000 per eye and uptake of the treatment has been steady, with sales of $15.6m in the first nine months of the financial year.
Toms told us that it is too early to share the price of the treatment in the EU and the UK, but suggested it may be necessary to ‘rethink’ the healthcare system’s approach to genetic diseases.
“It’s important to recognise that introducing one-time therapies, representing significant advances in the treatment of rare diseases, requires rethinking how our healthcare system manages diagnosis, treatment, care and associated costs for patients with genetic disease,” he continued.
Issues over pricing may lead to protracted discussions over what is deemed cost-effective by individual nations in Europe; Toms expects initial decisions to be announced by UK national authorities in 2019, but in a statement the company noted that it could be sometime in 2020 before patients are eligible to receive the treatment.
Cell and gene therapy focus
Investing in Luxturna has provided Novartis with access to a ground-breaking gene therapy treatment, to complement its work in the cell therapy space, which has seen the firm boost its manufacturing capacity for both areas.
Earlier this year, Novartis also acquired gene therapy specialist AveXis through an $8.7bn acquisition.