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Emil Kakkis
Chief Executive Officer and President at Ultragenyx Pharmaceutical Inc.
Emil Kakkis is a renowned figure in the field of rare disease treatment, with a strong background in biochemistry, medicine, and research. Throughout his career, he has successfully developed and obtained FDA approval for three medicines for rare disease patients, with an additional three in clinical studies.
Emil Kakkis is deeply committed to improving the regulatory process that governs the development of treatments for rare diseases. In 2010, he founded Ultragenyx Pharmaceutical Inc. where he currently serves as the Chief Executive Officer and President. Under his leadership, the company has embarked on multiple clinical programs, with four products and five clinical programs expected in the current year, boasting a team of approximately 70 professionals.
Specializing in drug development for rare diseases and biochemical genetic disease treatment, Emil Kakkis' expertise extends to the clinical management of mucopolysaccharidoses. His dedication to treating untreated rare diseases is evident in Ultragenyx Pharmaceutical Inc.'s mission to swiftly translate therapies for those in need.
Emil Kakkis pursued his education at the University of California, Los Angeles, where he studied MD/PhD in biological chemistry. He also holds a Bachelor of Arts (B.A.) in Biology from Pomona College.
In addition to his role at Ultragenyx Pharmaceutical Inc., Emil Kakkis has held significant positions in various organizations. He has served as the President at EveryLife Foundation for Rare Diseases, a Development Consultant at BioMarin, a Board Member at the National Organization for Rare Disorders, and Chief Medical Officer at BioMarin, among others. Earlier in his career, he was also an Assistant Professor at Harbor-UCLA Research and Education Institute as well as Harbor UCLA Med Center, Research and Education Institute.
Highlights
I met with Senior FDA leadership today with a group of rare disease patient advocates and experts to advocate for a change in policy to enable more accelerated approvals for precision medicines treating ultra rare diseases. The advocates and experts included including Mark Dant, Sharon King, Pam Andrews, Joseph Muenzer MD, Justin Hopkins MD, Christine Waggoner and on line Cara O’Neill MD, Elizabeth Berry-Kravis MD, Matthew Ellinwood DVM, Pat Furlong, and Terri Klein. We were honored and appreciative of the presence of the entire FDA leadership, including FDA Commissioner Rob Califfe, CBER director Peter Marks (online), Office of New Drugs head Peter Stein, CDER director Patrizia Cavazzoni, CDRH director Jeff Shuren, CMO Hilary Marston, CSO Namandje Bumpus, Office of Orphan Product Development OPD director Sandra Retzy, and Kristy Moran.
You cannot get a greater representation from FDA. We explained to them that we are in a serious crisis as many precision treatments for ultrarare diseases are getting terminated the last year and will be lost forever despite the fact they are already shown to be clearly working effectively in many cases and that the ability to get these treatments approved, especially if complex and neurologic, is essentially impossible in the current regulatory environment. We greatly fear that within the next two years, we may lose all of the new treatments for many diseases which seemed so promising just 2 years ago.
The current qualification of biomarkers is ineffective and not well managed by FDA at this time at the review levels despite the alignment at the senior levels. The FDA expressed their desire to collaborate on improving access to accelerated approval but mainly blamed companies for not doing all the work required. I know that this could be true for some smaller companies with limited resources but without any specific quality published guidance from FDA, how should they know the list of information they want? I do know specifically this is not the case for some applications that were complete and appropriate. The FDA is not yet doing what needs to be done with the urgency required and we will hold them to their word, that our framing of the problem on accepting biomarkers is sound, and require they take action now. Case by case, we will press them on the facts and press for this change. It is Time for a Change. Too many ultrarare disease patients are depending on us.
