Fabry Disease Patients Hope for FDA Approval of Drug Replagal


Genzyme Shortage of Fabrazyme Hurt Patients

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.

The company was forced to reduce patients to half doses, but in early August Genzyme stopped shipments altogether.

At the time, Bo Piela, vice president of corporate communications at Genzyme, said, "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."

The company is in the midst of constructing a second manufacturing plant in Framingham, Mass., and when that is approved, "we will be able to meet full demands," he said.

"But licensing takes time," he added.

Replagal and Fabrazyme came on the market at the same time, but the FDA gave exclusivity to the Genzyme drug, which has now expired. Shire went on to market its drug in Europe.

"We are coming up on 10 years experience with [Replagal]," said Shire's Gregoire. "We have lots of experience with the product. The reality is the population of Fabry patients in the U.S. has been suffering for two and half years."

Shire offered free treatment to 140 American patients -- about 20 percent of those with the disease -- under a protocol allowed ahead of approval for emergency use.

"We already have a fast-track designation," said Gregoire. "New patients will be able to get it when the product is approved. The standard time is six months, but the agency could do it in a shorter amount of time."

For Walter, whose family has seen 18 members affected by Fabry disease, the news is exciting. He has already exceeded the average age of death for an adult male with the disease, which is 50.

"In the bigger picture, we need treatment options and we can't rely on one medicine and be at risk of a drug shortage happening," said Walter, whose health has been "up and down."

"We have a life-threatening genetic disorder," he said. "The medication works, but it's not a cure."

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