Novartis in US$170m licence deal with Spark Therapeutics

The Swiss pharma major paid US$105m upfront for the commercialisation rights for the first-in-class gene therapy Luxturna outside the US market. Spark Therapeutics said it wants to sell the therapy for the rare inherited disease Leber's congenital amaurosis at an annual price of US$850,000 per patient. In December 2017, the company received FDA approval based on results of a Phase III study enroling 31 patients with the disorder. . Novartis said it will further pay a US$65m milestone upon European regulatory approval. Last July, Spark Therapeutics submitted an MAA to the European Medicines Agency. Spark will also receive royalties on net sales outside the US from Novartis, which will market the drug in Europe.

Leber's congenital amaurosis is a retinal dystrophy that affects 1,000-2,000 people in the US and „fewer than 52,000 people in Europe“, according to the EMA. It’s caused by approximately 300 biallelic mutations in the RPE65 gene, which expresses a protein that is essential for the sythesis of rhodopsin, the light-sensitive pigment found in the rods of the retina. However, other company’s that have received EU Orphan Drug Status in this indication target other genes, i.e. Pharma Gateway AB, Sweden, targets CEP290, or British MeiraGTx UK II Ltd targets the AIPL1 gene.

Luxturna is designed to be directly injected into the eye. The enzyme replacement therapy works by delivering viral vector particles containing a correct copy of the gene to retinal cells, restoring their ability to make the PPE65 enzyme. Compared to other enzyme replacement therapy Luxturna is set to double their average market price. So, it’s probable that the FDA will propose a value-based pricing scheme like in the previous case of Novartis’ cancer gene therapy Kymriah.

Spark said the price is US$425,000 "per eye". An ICER panel assumed a 10- to 20-year benefit of treatment for teenagers and concluded Luxturna would have to cost between US$152,612 and US$216,738 to meet standard cost-effectiveness. That's in the range of other enzyme replacement therapies on the market.