Emil Kakkis

Professional background

Kakkis began his work at Harbor-UCLA 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.

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 PhD 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.

Philanthropy

In early 2009, Kakkis founded the Kakkis EveryLife Foundation to accelerate biotech innovation for rare diseases. The Foundation initiated the CureTheProcess Campaign dedicated to improving the regulatory and clinical development process for rare diseases. The Campaign has been endorsed by more than 175 patient organization and physician society partners.

Kakkis spent the last year working with the U.S. FDA and Congress to improve the regulatory process for rare diseases. His goals were recognized in the passing of the Brownback Brown Amendment to the 2010 FDA appropriation bill. The bill requires 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).

Publications