BioMarin's five powerhouse 'orphan' drugs

BioMarin stories in this issue

BioMarin’s molecules yield miracles

BioMarin enzyme specialist speeds up science

BioMarin's five powerhouse 'orphan' drugs

BioMarin patient Kendra conquers MPS VI

SAN RAFAEL -- BioMarin Pharmaceutical took five drugs to market in about 13 years, an industry speed marvel.

Naglazyme, for MPS VI, was close to FDA approval when CEO Jean-Jacques Bienaime arrived at BioMarin. Aldurazyme, which treats MPS I, caused by lack of the alpha-L-iduronidase enzyme, was already on the market for about $200,000 a year, and is sold by Genzyme, based in Boston and acquired by Paris-based Sanofi in 2011. BioMarin considered a deal to have Genzyme also sell Naglazyme outside the United States.

Such a deal "would have been a big mistake," he said. "Probably BioMarin would not be independent today if we had done" such an agreement, as 85 percent of revenue from Naglazyme is from outside the U.S.

Vimizim, BioMarin's drug for MPS IV-A or Morquio syndrome, was approved in February 2014 and costs $380,000 a year.

A BioMarin drug for phenylketonuria or PKU is called Kuvan, approved in 2007. PKU is a birth defect that ruins the body's manufacture of the enzyme needed to break down phenylalanine, an amino acid. This is a larger market, with some 50,000 patients worldwide. BioMarin, which has reaped total estimated Kuvan sales of $750 million, faces a potential generic threat from an unnamed company that filed a patent challenge with the FDA earlier this year.

The fifth BioMarin drug, on the market so far only in Europe, is Firdapse, which treats Lambert-Eaton Myasthenic Syndrome, an autoimmune neuromuscular disease that results in weakness.Cancer drug pioneers

In 2010, BioMarin bought San Bruno-based LEAD Therapeutics for about $97 million to acquire its oral oncology drugs, especially LT-673, for genetically caused cancers.

"Every tumor has a specific genetic profile that you can use as you attack the cancer," Mr. Bienaime said.

Show Comment