StemCell Therapy

BioMarin Pharmaceutical on Monday dedicated its new Novato manufacturing facility which is expected to be key in its continuing clinical trials on a drug the company believes will potentially genetically repair the cause of hemophilia A.

Before a crowd of 300 to 400 people, the company, which manufactures drugs mostly for rare diseases, called its new production location the largest gene-therapy-manufacturing facility in the world. The project was completed 11 months ahead of schedule, employing 300 people in 200,000 construction hours, according to Robert Baffi, the firms executive vice president, Technical Operations.

Jean-Jacques Bienaim, chairman and CEO of BioMarin, said the drug to be produced at the location, BMN 270 gene therapy for hemophilia A, has the potential to change what future doctors learn about hemophilia.

Because of a genetic flaw, the blood of those who have hemophilia does not clot. The mutation takes places in a single gene that provides instructions to make a protein called Factor VIII, which is essential for blood to clot normally.

According to the company, the drug thus far in investigational clinical trials has shown the ability to genetically correct the problem and allow patients to manufacture and maintain a constant level of Factor VIII. Production of the drug to be used in those continuing trials will begin as soon as possible in Novato.

Among those affected by the hemophilia is the son of Christine Orr a speaker at todays event. Genetic roulette resulted in an older son being born without the problem.

But soon after her younger son was born, it became apparent he had little or no clotting factor. Every other day, home infusions of clotting factor have helped curb the problem, but she said her son experienced the stigma of parents being afraid to invite him to birthday parties or play dates over what might happen if he were to be hurt.

She said a one-shot treatment to potentially genetically treat and cure the disease gives her hope that yes, a cure is on my horizon, and he can choose his path in life and not have hemophilia choose it for him.

On Aug. 2, BioMarin Pharmaceutical reported it reaped $317 million in second-quarter revenue, up 6 percent from the same quarter in 2016.

It operated a loss of $37 million for the second quarter, but far less than the $419 million loss in the same quarter last year. The last quarters losses amounted to 21 cents per diluted share.

BioMarin, which has six main drugs on the market, had two huge contributors to second-quarter revenue: Kuvan, with $102 million, and Vimizim, with $103 million.

Kuvan, sapropterin dihydrochloride, treats a genetic disorder called phenylketonuria. BioMarin bought global rights to Kuvan in 2015 from Merck for 340 million euros, about $405 million. PKU is rare, and causes amino acid phenylalanine to build up in the body. The buildup of the amino acid can cause grave health problems.

Vimizim treats patients with mucopolysaccharidosis type IV-A, also called Morquio A syndrome, which is a metabolic disorder that inhibits the bodys ability to process certain mucopolysaccharides. It is usually inherited.

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BioMarin Pharmaceutical launches gene therapy drug plant in Novato - North Bay Business Journal

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