FDA's minding is to give more options to patientsLook at the news below. You'll understand how the FDA thinks. It is there to spur innovation, give additional options to patients.
And then, consider what TLT's treatment could offer to its own pool of late-stage patients; a possibility to save their bladder. Again, our treatment has no SAE and can avoid/delay a bladder removal, have a much better quality of life. So why would the FDA remove such a new alternative to patients.
Change this indication below and replace it by BCG-unresponsive that could lead to cystectomy and see what I underscored in the article below and you'll see that it could apply to our drug.
Hemgenix is a gene therapy to treat adults with hemophilia B, a genetic bleeding disorder in which people do not produce a protein needed to create blood clots. About 1 in 40,000 people have the disease, most of whom are men. Typical treatment requires routine intravenous infusions to maintain sufficient levels of the missing or deficient clotting factor, but the new gene therapy is meant to be a one-time IV infusion.
“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Today’s approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.”
The Institute for Clinical and Economic Review, an independent nonprofit research organization that analyzes the value of prescription drugs and other medical products, confirmed for CNN that Hemgenix is now the most expensive drug in the world.
In a recent cost-effectiveness analysis of the drug, weighing health benefits against offset costs, ICER suggested that a fair price for the drug to be between $2.93 million and $2.96 million.