Here are four things to know.
1. Luxturna is a one-time gene therapy intended to treat about 1,000 to 2,000 Americans with the inherited retinal disease Leber congenital amaurosis.
2. The FDA acknowledged Luxturna’s short-term efficacy, but said long-term clinical benefits are unknown, since no data exists on the gene therapy’s efficacy after a year.
3. If approved, Luxturna would be the first gene therapy used to treat an inherited disease in the U.S.
4. Spark Therapeutics CEO Jeffrey Marrazzo declined to comment on the future price of the drug during an interview with The New York Times. However, he did say the company will consider the price of other rare disease drugs, such as treatments for Pompe disease and Hunter syndrome, when setting a price for Luxturna. Many of these drugs cost $300,000 to $600,000 annually.
More articles on supply chain:
Express Scripts to acquire eviCore for $3.6B: 4 things to know
This startup will deliver defibrillators to 911 callers via drone
4 drug, devicemakers in the headlines
At the Becker's 11th Annual IT + Revenue Cycle Conference: The Future of AI & Digital Health, taking place September 14–17 in Chicago, healthcare executives and digital leaders from across the country will come together to explore how AI, interoperability, cybersecurity, and revenue cycle innovation are transforming care delivery, strengthening financial performance, and driving the next era of digital health. Apply for complimentary registration now.
