Novartis' unique gene therapy calls for "rethinking" the costs of health

In January of this year, Novartis bought Spark Therapeutics' ex-US rights to Luxturna (voretigene neparvovec-rzyl) for $ 105 million (€ 92.7 million), to which an additional 65 million payments were added. of stage potentially due.

Spark has received approval from the US Food and Drug Administration at the end of last year, the treatment becoming the first gene therapy available for the treatment of patients with genetic disease . It has now received the approval of the European Commission for the treatment of the same condition.

Luxturna is a single-use therapy designed to restore functional vision in children and adults with biallelic mutations of the RPE65 gene.

Mark Toms, CSO of Novartis UK, described how the treatment works: "[Luxturna] provides a functional copy of the RPE65 gene to act in place of the mutated RPE65 gene. This functional gene produces the RPE65 protein, which can prevent the progression of blindness in patients with RPE65 by improving visual acuity. "A flawless stay

PriceA flawless stay

In the United States, Spark values ​​the product at $ 425,000 per eye and the use of treatment is stable, with sales of $ 15.6 million in the first nine months of the fiscal year.

Toms told us that it was too early to share the price of treatment in the EU and the UK, but suggested that it may be necessary to "rethink" the health system's approach to health care. genetic diseases.

"It is important to recognize that the introduction of timely therapies, which represent significant advances in the treatment of rare diseases, requires rethinking how our health system manages the diagnosis, treatment, care and associated costs for patients with genetic disease, "He continued.