Emil Kakkis | |
|---|---|
| Born | 1960 |
| Education | Pomona College UCLA David Geffen School of Medicine at UCLA |
| Known for | Developing treatments for ultra rare disorders |
| Medical career | |
| Institutions | Ultragenyx Pharmaceutical Inc., Founder, CEO and President Kakkis EveryLife Foundation, Founder BioMarin, Former Chief Medical Officer |
| Sub-specialties | Rare biochemical and genetic diseases also known as inborn errors of metabolism |
| Research | Enzyme Replacement Therapy |
| Awards | Bogen Prize Vaile Prize in Biology Lifetime achievement award from the National MPS Society California Life Sciences Award |
Emil Kakkis (born 1960) is an American medical geneticist known for his work to develop treatments for ultra rare disorders. He is the Founder of the Everylife Foundation for Rare Disease and Founder, CEO and President of Ultragenyx Pharmaceutical Inc.
Kakkis began his work at Harbor–UCLA Medical Center working with minimal funding and support to develop an enzyme replacement therapy (Aldurazyme) for the rare disorder Mucopolysaccharidosis (MPS I). The struggle to get the therapy translated from a successful canine model to patients succeeded due to the critical financial support of a patient organization formed by Mark and Jeanne Dant for their son Ryan, called the Ryan Foundation.[1]
Aldurazyme development was later supported by BioMarin and eventually, their partner Genzyme leading to U.S. Food and Drug Administration (FDA) approval in 2003. During his tenure at BioMarin, Kakkis guided the development and approval of two more treatments for rare disorders, MPS VI and PKU and has contributed to the initiation of seven other treatment programs for rare disorders, three of which are now in clinical development.
Kakkis is board certified in both Pediatrics and Medical Genetics. He graduated from Pomona College, magna cum laude, received combined MD and Ph.D. degrees from the UCLA Medical Scientist Program, and received the Bogen prize for his research. He completed a Pediatrics residency and Medical Genetics Training Fellowship at Harbor-UCLA Medical Center. He became an assistant professor of Pediatrics at Harbor-UCLA Medical Center from 1993 to 1998 where he initiated the enzyme therapy program for MPS I.[2]
In early 2009, Kakkis founded the EveryLife Foundation to accelerate biotech innovation for rare diseases. The Foundation initiated the CureTheProcess Campaign to improve the regulatory and clinical development process for rare diseases. The Campaign was endorsed by more than 175 patient organization and physician society partners.[3]
Kakkis spent the following year, 2010, working with the U.S. FDA and Congress to improve the regulatory process for rare diseases. This resulted in the Brownback Brown Amendment to the 2010 FDA appropriation bill.[4] that required the FDA to review its rare disease regulatory policies and look for ways to improve. The FDA is working on a report to Congress and plan for improvements by the deadline of September 2011.
The Kakkis Family and the Foundation are major supporters of projects that help the rare disease community such as RareArtist.org, EveryLife Art Contest, Global Genes Project, National MPS Society, Rare Disease Legislative Advocates and the SIMD’s North American Metabolic Academy (NAMA).