Genzyme Drug Shortage Leaves Users Feeling Betrayed

Carol Fink of Yountville, Calif., says lack of the medicine she needed left her constantly in pain, sapped her energy and made her thinking fuzzy.

For Dr. William Schubert, an obstetrician and gynecologist in Pocatello, Idaho, the factory shutdown may have contributed to an even worse outcome. Even as his wife and doctor raced to find doses of the drug he needed, Dr. Schubert’s heart deteriorated rapidly. He died on March 6, at the age of 63.

These people, and thousands more in this country and abroad, have been hard hit by a shortage of drugs made by the biotechnology company Genzyme to treat two rare inherited diseases.

The supply problems, which have dragged on since last summer, have frayed the close relationship — unusual in the pharmaceutical industry — that Genzyme had carefully built with the several thousand users of its high-price medicines over the last two decades.

Some of those patients now say they feel betrayed by the company they once viewed as their savior, wondering why Genzyme did not have a sufficient reserve of such vital drugs and how the company could have stumbled so badly in trying to fix its production problems.

“If most businesses run like this they’d be out of business,” said one patient, Mark Malone of Morris, Ill. “Unfortunately, they have the drug we need.”

Last June the company temporarily shut its main factory in Boston because of contamination from a virus. Such problems can arise in biotechnology factories, which use living cells to make drugs, and few faulted the company at the time.

But Genzyme, which initially predicted that the drug shortages would last six to eight weeks, has repeatedly backtracked on when supplies would be fully restored, as it has run into further manufacturing problems. At one point, particles of steel, rubber or fiber were found in some vials of the drugs.

The repeated setbacks have hurt the earnings and stock price of Genzyme, one of the five largest biotechnology companies, with $4.5 billion in sales last year. The company’s adjusted earnings per share fell 26 percent in the fourth quarter of 2009, though they were still up 16 percent for the year. The stock which traded above $72 early last year, closed Thursday at $52.77.

The production problems have also left the company open to a proxy challenge from the investor Carl C. Icahn. The company has also said it was likely to be fined by the Food and Drug Administration.

Beyond the financial costs, though, is the toll on people whose health depends on those two drugs: Cerezyme, for Gaucher disease, and Fabrazyme, used against Fabry disease.

Both diseases are rare inherited enzyme deficiencies that allow fatty substances to build up in the body, damaging organs. The Genzyme drugs, which are typically given intravenously every two weeks, provide the missing enzyme for each disease.

Companies that sell pills for widespread conditions like diabetes or depression rarely know the identities of the people who use their products. But Genzyme knows virtually all the patients, at least in the United States.

That is in part because there are only 1,500 Cerezyme users and fewer than 1,000 Fabrazyme users in this country. It is also because the drugs are so expensive — about $200,000 a year. Many patients turn to a Genzyme case worker to help them secure insurance or financial aid.

After Dr. Schubert was found to have Fabry disease five years ago, he and his wife sold their house and bought a smaller one to help them afford the nearly $4,000 a month in premiums their insurer began charging them to cover the drug. “We had invested everything in that, emotionally and financially,” his widow, Janet Schubert, said.

Henri A. Termeer, chief executive of Genzyme, acknowledged that the company had let patients down. “We have this enormous humility,” he said in an interview. “We have to re-earn our standing with these patients.”

Mr. Termeer said that when the viral contamination occurred, the company had unusually low reserves of Cerezyme and Fabrazyme because it was temporarily using its Boston factory to produce a third drug, one for another rare disease, Pompe, which is often fatal to the children it afflicts.

“Many patients’ lives were saved” by the Pompe drug, Mr. Termeer said. “Given that we had never had a virus before,” he said, referring to the factory contamination, “it was probably an understandable decision. But it was a high price to pay.”

Genzyme now says full supplies of Cerezyme will be available May 1, and of Fabrazyme perhaps in the third quarter.

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via nytimes.com

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