Emil Kakkis, MD, President and Founder of Ultragenyx Pharmaceutical Inc., explains that while the Orphan Drug Act of 1983 may have been the catalyst to begin developing drugs for rare diseases, other factors are now in place to make orphan drug development a viable medical and business venture.

According to Dr. Kakkis, most believe that the Orphan Drug Act was the catalyst for rare disease drug development. While he agrees the law helped facilitate this development, he argues that it is not necessarily responsible for the current funding given to companies like Ultragenyx and others that are devoted to developing treatments for rare and ultra-rare diseases. Instead, he points to the success of early rare disease treatments like alglucerase (Ceredase) for Gaucher disease, as a reason to give investors confidence in funding future rare disease drug development. 

Along with the Orphan Drug Act and increased interest in funding rare disease treatment, Dr. Kakkis asserts the important role technology has played in treatment development. Not only has new technology broadened the possibilities for different types of therapies, it has allowed researchers to develop treatments for diseases that were previously seen as untreatable. 

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