NOVATO -- A highlight of Friday morning's ground-breaking ceremony for BioMarin Pharmaceutical's 85,000-square-foot new research-and-development laboratory at the San Rafael headquarters campus was the emotional testimony of a patient in one of the company's clinical trials who said the treatment for his rare disease helped him avoid debilitating developmental mental and other conditions.
In front of the cleared ground where construction crews plan to build the three-story lab building, Bailey Fleming told dozens gathered for the occasion that BioMarin contacted in 2005 about participating in a clinical trial of Kuvan to treat his phenylketonuria, commonly known as PKU. It's a hereditary condition that prevents babies from metabolizing the amino acid phenylalanine, which is found in foods with protein.
Because of the disease, that amino acid and related substances build up in the body, deteriorating the central nervous system, often leading to brain damage.
Mr. Fleming and others with PKU are assigned an extremely low-protein diet. His participation in the clinical trial of Kuvan contributed to its approval in the U.S. in 2007 and elsewhere the following year.
Now he gets to eat regular bread and pasta and is going to school with dreams of becoming an airline pilot. Perhaps, one day he will be able to eat a steak, he told the audience.
"None of these dreams would have been possible without that drug," he said.
Robert Baffi, BioMarin executive vice president of technical operations, told the gathering that the project will result in hundreds of construction jobs while the lab and a 650-space parking garage are built. Ultimately, 300 researchers will work in the building, joining 300 already working in two buildings of the soon-to-be five-building San Rafael Corporate Center campus. [See "BioMarin starts new San Rafael buildings," Aug. 1.]
BioMarin will continue to have more than 500 working from the Novato production campus, he said.
Mayor Gary Phillips said he hopes the forthcoming Sonoma Marin Area Rail Transit train, which will serve San Rafael and Novato along the line, will be an attraction for BioMarin. The company started its headquarters move to San Rafael in 2012.
"We want to welcome you to San Rafael once again," he said.
Assemblyman Marc Levine, who was on the City Council when BioMarin applied to change the San Rafael Corporate Center zoning to allow a lab, said BioMarin's work with rare diseases has made an impact on fellow legislators in Sacramento. Rep. Jared Huffman, former occupant of that Assembly seat, and Mr. Levine put forward a state resolution to declare each Feb. 28 Rare Disease Day.
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