Fabry Disease Patients Get Sicker as Drugs Go Overseas

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Their lawyer, C. Allen Black of Pittsburgh, said many more will soon be joining the litigation.

"This is one of the worst avoidable tragedies in American medicine, and the suffering has been incalculable," according to Black, who said patients are suing for millions of dollars in damages because they are so sick they cannot work. The drugs cost $30,000 for each infusion, and a full dose consists of two infusions a month.

Genzyme's problems began in June 2009 when the only plant that manufactured the biological drug was shut down in Allston, Mass., by the FDA because of virus contamination

The company was fined $175 million and issued a consent decree to remediate. The French pharmaceutical giant Sanofi-Aventis took over the company in April.

Genzyme admitted to miscalculations in its drug reserves, which caused it to cut back on supplies of Fabrazyme to patients, but it said the shortage would eventually be resolved.

"People who work at Genzyme feel a close connection with patients," said Bo Piela, vice president of corporate communications at the pharmaceutical company. "We understand the impact that the Fabrazyme shortage has had on patients and their families. Our highest priority is to restore full supplies of the medicines we make. We are doing everything possible to achieve this and are making good progress toward the approval of a second manufacturing plant that will allow us to significantly increase product supply."

A similar drug, Replagal, which is marketed in Europe by the biopharmaceutical company Shire,

has not been approved for sale in the United States. About 100 Americans with Fabry disease are receiving Replagal through an investigational clinical trial sanctioned by the FDA

"Unfortunately, Shire withdrew its application for FDA approval [of Replagal], so, there is not much we can do to force Shire to make it available in the U.S.," said Black. "The only solution for U.S. patients is to increase the production of Fabrazyme."

Debra Lewis, acting director of the FDA's Office of Orphan Products Development, said the agency gives exclusive marketing to drugs for seven years, and Genzyme's rights to Fabrazyme expired in April 2010. "As far as the FDA is concerned, it would not limit another product," she said.

"We would welcome working with an outside company to help address the shortage," said Lewis. "We have limits to our authority, and people don't want the FDA to go beyond its charge to see if a product is safe and effective."

In the meantime, patients like Walter report a steep decline in their health -- heart enlargement, kidney failure and neurological problems. They are also worried about reports from Europe of health risks associated with reducing medication.

In a 2010 letter from Genzyme Europe, the company said that lowered dosages of the drugs could cause "aggravation" of disease symptoms and or "adverse events" -- "pain, cardiac manifestations and deafness."

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