Dive Brief:
- "We recognized the importance of the trail we are blazing and have approached the commercialization of Luxturna with this in mind," said Spark Therapeutics Inc. CEO Jeffrey Marrazzo about the launch of its gene therapy for a genetic form of blindness during a Feb. 20 earnings call with analysts.
- While awaiting launch of Luxturna in the U.S., Spark is still angling for approval in Europe. The company said it expects regulatory action in the third quarter and that EMA good clinical practice audits for Spark and its sites have been completed.
- Last month, Spark announced that it has partnered with Novartis AG to commercialize the gene therapy outside the U.S. "We believe Novartis' existing infrastructure and capabilities will result in a broader and timelier rollout to patients outside of the United States, and importantly, outside of Europe, potentially increasing the value of this strategically important therapy," added Marrazzo.
Dive Insight:
As the first company to get a true gene therapy approved in the U.S., investors and analysts are watching Spark's roll out of Luxturna (voretigene neparvovec-rzyl) closely. The drug is expected to hit the market late in the first quarter.
The company already made waves when it announced its innovative approach to pricing the one-time therapy. It has put in place several programs to allow for patients to pay over time and for payers to get reimbursed if the therapy doesn't live up to efficacy and durability claims.
Now, the market is watching to see how the drug will perform and if Spark is taking precautions to make the commercialization effort a success. Spark has lofty barriers to break down for the one-time therapy; Gene therapies that were launched in Europe largely flopped.
John Furey, Spark's chief operating officer, laid out the four key initiatives that are driving the launch of Luxturna, during the fourth quarter earnings call on Tuesday.
He identified the first initiative as finding the appropriate patient. "Our prelaunched ID YOUR IRD initiative was well received and a follow-on initiative offering a free single gene test of RPE65 is underway. Additionally, our medical and diagnostic teams are educating health care providers about the importance of identifying and genotyping patients with inherited retinal diseases," he said, noting the company has fully staffed a patient services hub to help with questions regarding testing.
The second initiative focuses on treating the appropriate patient. The company is currently using its medical liaison team to reach out to doctors and previously diagnosed patients in order to get Luxturna to those patients. Furey estimates that anywhere between 50% to 70% of already diagnosed patients could be eligible for the gene therapy.
The third launch initiative is geared toward the patient experience. Spark has picked seven treatment centers across the country where the one-time therapy will be given. The biotech is already training doctors and pharmacists at those sites, as well as providing patient support groups that can help with insurance paperwork and other cost issues.
The fourth priority is to continue to work with payers to set up similar agreements to the ones already in place.
"With respect to the launch, we are tracking to our internal metrics and are confident that we have the right expertise and capabilities in place to execute across these four areas of focus. Because of the uniqueness of this treatment and commercial launch, we will gain clarity on launch metrics as we progress throughout the year," the COO added.