Advancements in the work with genes at Spark Therapeutics has led to what appears to be a cure for a specific type of blindness. Retinal dystrophy is the blindness that occurs when an individual has inherited a mutated RPE65 gene from both of their parents. The cure for this blindness has been discovered and will cost the patient $425,000 per eye amounting to grand total of $850,000.
The cure developed by Spark Therapeutics is known as Luxturna. Approved by the United States Food and Drug Administration, Luxturna is a one-time drug treatment that goes directly after the origin of retinal dystrophy. The drug repairs the mutated gene and DNA directly and then allows for the body to correct the mutation and the vision impairment by itself. The one-time gene therapy does not guarantee full vision to be restored to all patients, but has proved to be very successful in its clinical trial. This drug is the very first gene therapy to be approved by the U.S. FDA
The cost of the Luxturna treatment is quite shocking to some, but to others in the industry, the cost of $850,000 is a sensible cost for the return of one’s vision. The drug was expected to cost closer to one million dollars. When deciding on the price of a medical treatment like this, there are a few factors to consider. One of those factors being the number of patients that can benefit from the medicine. At the time of its development there were a thousand individuals suffering from retinal dystrophy in the United States and a prediction of 20 additional cases yearly. That small population of patients is believed to be a large reason behind the $850,000 price tag.
Spark Therapeutics has been working with health care providers to give rebates to patients that are not successful with the Luxturna treatment following an allotted amount of time. Some of their negotiations include allowing patients a payment plan that last over years. Luxturna is the most expensive drug treatment in the world and Spark Therapeutics is working to ensure that patients that need it are able to get it.