By Ethan Whiteford
Recently, Bloomberg published an article highlighting the FDA process of approving a treatment created by the company, Sarepta Therapeutics Inc. The approval has sparked fierce debate within the Food and Drug Administration. Sarepta’s drug is called Exondys 51 and it is used to treat Duchenne muscular dystrophy, which is a rare, genetic disease that essentially “wastes” muscles. It happens to boys in their youth and it kills within years. Until recently, there was no cure. Exondys 51 will cost $300,000 a year after discounts. FDA Director Robert Califf expressed caution about reading too much into the decision. He is quoted saying: “Our understanding about how to include patients in the regulatory process is evolving. Serious shortcomings in the eteplirsen (the drug that Exondys 51) is development program should not be allowed to establish broad precedent for therapeutic development in rare diseases.” The FDA’s chief scientist, Luciana Borio, said that if Exondys 51 did not get accelerated approval, Sarepta would not have sufficient funding to continue to study eteplirsen.
This relates heavily to what we talked about last Tuesday. We talked about profiting from health and this article is a perfect example of the debate going on currently about drug prices in the US. On one side, you have the patients and their families being unable to afford such a high price for treatment. And on the other side, you have the company, which without the price being so high, are unable to provide the life-saving drug and are also unable to research it to make it better