Healthy Skepticism Library item: 18577

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Publication type: Electronic Source

Pollack A
Patients Want Patent Broken on Genzyme Drug
The New York Times Health Blog 2010 Aug 2
http://prescriptions.blogs.nytimes.com/2010/08/02/patients-want-patent-broken-on-genzyme-drug/

Full text:

Updated: Three patients with a rare genetic disease are asking the federal government to abrogate the exclusive patent rights held by the biotechnology company Genzyme on the only drug now approved to treat the illness.

Because of manufacturing problems, Genzyme has not been able to produce enough of the drug, called Fabrazyme. The drug is used to treat Fabry disease, an inherited enzyme deficiency that can cause heart and kidney problems, as well as pain and other symptoms.

Fabry patients have been receiving only about one-third of their usual doses, and many say they are experiencing increased pain, gastrointestinal problems, and other symptoms. At least one patient might have died from heart problems exacerbated by the drug shortage.

In their petition, the patients urge the Department of Health and Human Services to exercise the government’s so-called march-in’ rights to force the patents covering Fabrazyme to be licensed to other manufacturers to help alleviate the shortage.

The government has such a right, the petition says, because the National Institutes of Health financed the research at the Mount Sinai School of Medicine in New York that led to the drug’s development. Mt. Sinai gave an exclusive license to Genzyme.

“It is unreasonable, improper and even catastrophic to limit patient access to a drug where such a limitation causes morbidity and death,’’ the petition states. “Limiting access instead of encouraging others to make up the shortfall in manufacturing is the worst conceivable public health solution to supply shortages of publicly funded inventions.’’

The petition proposes that Genzyme be paid a 5 percent royalty on sales by the other manufacturers. Bo Piela, a spokesman for Genzyme, said the company could not comment immediately because it was not aware of the petition. But he said the company was making progress in fixing its manufacturing problems and hoped to be increasing the supply of Fabrazyme in the fourth quarter of this year. A new manufacturing plant is expected to be in operation late next year to ensure adequate supply, he said.

The petition was filed by Joseph M. Carik of North Las Vegas, Nevada; his cousin Anita Hochendoner of Pittsburgh; and her daughter Anita Bova, also of Pittsburgh. They are being represented by C. Allen Black, a lawyer in Pittsburgh, with the support of Knowledge Ecology International, a Washington advocacy group that has worked to limit patent rights that it views as interfering with public health.

The request appears to be an uphill battle. According to the petition, there have been three previous requests for march-in rights on other drugs, and all were rejected.

But James Love, director of Knowledge Ecology International, said that in two other cases – involving human embryonic stem cells and a technique for making flu vaccines – federal pressure led to more liberal patent licensing without the need to resort to march-in rights.

Another question is whether granting such rights would even help alleviate the shortage. Given the time it takes to develop and test a drug, it is unlikely another supplier of Fabrazyme could be ready by late next year.

A nearer-term alternative is Shire’s drug Replagal, which has long been approved in Europe to treat Fabry disease. Shire is trying to get approval in the United States. In the meantime, given the shortage of Fabrazyme, American patients have been able to get Replagal on an experimental basis.

But now Shire’s supply is strained as well, so it is no longer accepting new patients in the United States.

Shortages of Fabrazyme as well as of Cerezyme, a drug for another rare illness called Gaucher disease, have hurt Genzyme’s earnings and stock price, leaving the company vulnerable to being taken over. Sanofi-Aventis is said to be preparing a bid.