Date: 2018-01-24
Type of information: Licensing agreement
Compound: Luxturna™ (voretigene neparvovec-rzyl - SPK-RPE65)
Company: Spark Therapeutics (USA - PA) Novartis (Switzerland)
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Type agreement: licensing
Action mechanism:
- gene therapy. The RPE65 gene provides instructions for making an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision. Individuals with biallelic RPE65 mutation-associated retinal dystrophy experience progressive deterioration of vision over time. This loss of vision, often during childhood or adolescence, ultimately progresses to complete blindness.
- Luxturna® works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient’s vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.
Disease:
Details:
- • On January 24, 2018, Novartis announced a licensing agreement with Spark Therapeutics covering development, registration and commercialization rights to voretigene neparvovec in markets outside the US. Voretigene neparvovec, known as Luxturna™ (voretigene neparvovec-rzyl) in the US, received FDA approval on December 19, 2017 as a one-time gene therapy to restore functional vision in children and adult patients with biallelic mutations of the RPE65 (retinal pigment epithelial 65 kDa protein) gene.
- The market authorization application (MAA) with the European Medicines Agency (EMA) was filed on July 31, 2017. Currently there is no existing therapy for this disease outside the US.
Financial terms:
- Novartis will make an upfront payment as well as pay milestones and royalties to Spark Therapeutics reflective of the late stage of the opportunity. Spark Therapeutics retains exclusive rights for Luxturna™ in the US and will retain responsibility for obtaining EMA approval. Commercialization rights will be transferred to Novartis upon successful completion of registration and issuance of market authorization. Novartis has exclusive rights to pursue development, registration and commercialization in all other countries outside the US. Spark Therapeutics will be responsible for the supply of voretigene neparvovec worldwide under a separate manufacturing and supply agreement with Novartis.
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