Novartis reached an agreement worth potentially more than $170 million to develop and commercialise Spark Therapeutics' gene therapy Luxturna (voretigene neparvovec) in markets outside the US, the companies announced Wednesday. Luxturna is currently under review in Europe for the treatment of patients with vision loss due to Leber congenital amaurosis or retinitis pigmentosa caused by confirmed biallelic RPE65 mutations. Meanwhile, Spark retains exclusive rights for Luxturna in the US, where it was approved last month to restore functional vision in children and adults with biallelic mutations of the RPE65, with Novartis noting that there is currently no therapy for the disease available outside the country.
Under the agreed terms, Novartis will make an upfront cash payment of $105 million to Spark, which is also eligible for up to an additional $65 million in cash milestones based on near-term European approval and initial sales in select markets outside the US. Further, Spark is entitled to receive royalties on net sales of Luxturna outside the US, if approved.
Meanwhile, Spark continues to be responsible for obtaining European approval of the gene therapy, with commercialisation rights to be transferred to Novartis after the successful completion of registration and market authorisation. Spark has also agreed to manufacture and supply Luxturna to Novartis under a separate agreement with the Swiss drugmaker.
Shreeram Aradhye, global head of medical affairs and chief medical officer at Novartis Pharmaceuticals, remarked "this collaboration builds on our commitment to ophthalmology." Aradhye added "we look forward to leveraging our global presence to ensure that patients outside the US have access to this potentially life-changing treatment."
Earlier this month, Spark revealed that it planned to price Luxturna at $425 000 per eye, or $850 000 in total, in the US. The company also said at the time that it had reached an agreement in principle with health services company Harvard Pilgrim to make the one-time gene therapy available under an outcomes-based rebate programme, while a proposal to the US Centers for Medicare & Medicaid Services would see Luxturna payments made over time.
For related analysis, see ViewPoints: One big step for Spark, one (potentially) giant leap for high-priced drugs and ViewPoints: Spark's payment plans reassure investors about Luxturna access. See also, Spotlight On: Gene therapy companies open up about pricing strategies.
Separately on Wednesday, Novartis announced that its fourth-quarter sales climbed 5 percent year-over-year to $12.9 billion, topping analyst estimates of $12.8 billion, lifted by the performance of new drugs such as Cosentyx and Entresto.
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