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Spark unveils 2018 plans at health care conference
Company co-founder and CEO Jeffrey D. Marrazzo calls 2017 a 'trailblazing year.'
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In 2018, Spark Therapeutics will work toward the continued expansion of its recently approved Luxturna treatment, company co-founder and CEO Jeffrey D. Marrazzo said in a presentation at the 36th Annual J.P. Morgan Healthcare Conference.
With Luxturna’s FDA approval, Marrazzo described 2017 as a “trailblazing year for us at Spark.” Luxturna (voretigene neparvovec-rzyl) is a gene therapy approved to treat children and adults with confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells.
Spark Therapeutics ended 2017 with $539 million in cash, cash equivalents and marketable securities. It also received a priority review voucher regarding Luxturna’s FDA approval, he said.
Luxturna
Luxturna will likely be available at the end of the first quarter, and treatments are expected to begin in the second quarter. European approval for the drug is progressing as well, Marrazzo said, with a regulatory decision expected in the third quarter.
“We are expecting to see patients treated [in Europe] in 2019,” he said.
The FDA’s approval of a “broad label” for Luxturna was also a high point of 2017, he said. The label describes the therapy’s safety profile but also includes a “genetic indication,” which provides for patients to be confirmed by a genetic diagnosis without “regard to the phenotypic diagnosis, which is not only important for Spark, but for many diseases the entire genetic field may be working on,” he said.
The label does not include an upper age limit and recommends treatment for appropriate patients 12 months of age and older.
“The FDA was interested in ensuring that physicians were able to use their discretion in determining who was clinically eligible,” Marrazzo said.
Payment methods
Spark Therapeutics is exploring a three-pronged approach to address the cost of the therapy. First, a new contract model would allow the company to sell Luxturna to the payer, either directly or through a specialty pharmacy. Second, the company would offer an outcome-based rebate arrangement for patients at 30 to 90 days and 30 months. If the designated outcomes are not reached at these dates, Spark would offer rebates to patients not exceeding the standard Medicaid rebate, Marrazzo said.
“The third principle is to work toward a solution that can allow for payments to be made in installments over time. We believe this is an important principle to work toward, not just for Luxturna,” he said, adding that such a plan would be applicable “in general for gene therapy.”
New candidate for choroideremia
Marrazzo said interim data were released for 15 participants in an ongoing phase 1/2 trial of the ocular gene therapy candidate SPK-7001 for choroideremia. No serious adverse events were reported. An additional cohort of five patients was enrolled at an earlier stage of the disease, he said.
Additional analyses will be released this year, he said. – by Robert Linnehan
- Reference:
- Marrazzo JD. Spark Therapeutics Inc. Presented at: 36th Annual J.P. Morgan Healthcare Conference; Jan. 8-11, 2018. San Francisco.
- For more information:
- Jeffrey D. Marrazzo can be reached at Spark Therapeutics, 3737 Market St., Philadelphia, PA 19104.
Disclosure: Marrazzo reports he is the co-founder and CEO of Spark Therapeutics.