Genzyme drug sees new supply disruption
Patients may have to miss doses after batch fails to meet standards
Globe Staff / March 24, 2011
Set to turn over its business to a new owner this spring, yesterday told patients who take its Fabrazyme drug that they may have to delay or miss one or more doses over the next few months because of a new supply disruption at its Allston Landing plant.
Workers at the manufacturing site had to reject a batch of Fabrazyme — a treatment for the rare genetic disorder Fabry disease, which causes a dangerous build-up of tissue fat — because of “environmental monitoring findings,’’ saidGenzyme spokeswoman Lori Gorski.
Gorski said Genzyme, which has its headquarters in Cambridge, wouldn’t specify the size of the Fabrazyme batch or elaborate on the nature of the environmental findings except to say the lot failed to meet industry standards for manufacturing procedures.
“This is clearly disappointing,’’ Gorski said, noting the company has been rationing Fabrazyme because of a viral contamination at the same plant nearly two years ago. “But we’re vulnerable to these disruptions because we’re operating with such limited inventory.’’
The latest supply disruption took place in the plant’s “fill-finish’’ suite during the last stage of the manufacturing process. Employees working in fill-finish load vials with Genzyme’s protein-based biotechnology drugs, freeze-dry them, cap them, and ship the products to health care providers worldwide. Gorski said the disruption did not affect production of Fabrazyme and another biotechnology drug, Cerezyme, both of which are grown from living cells inside bioreactors in the same plant.
“This issue only concerned the fill-finishing suite,’’ Gorski said yesterday. “Production is continuing to go well.’’
She noted that Genzyme is closing the fill-finish suite in Boston. It recently moved its fill-finish operation for Cere zyme to a company site in Waterford, Ireland, and is in the process of turning over fill-finishing for Fabrazyme and other Genzyme drugs to an outside contractor, Hospira, which has operations in McPherson, Kan.
Jack Johnson, a Fabry disease patient and executive director of the Fabry Support and Information Group in Concordia, Mo., said patients who take Fabrazyme are receiving a half-dose every two weeks — half of what they had been taking before the virus was found in June 2009. Some patients have experienced fatigue, pain, or gastrointestinal symptoms as a result, he said.
“At this point, I’m uncertain how serious this is,’’ Johnson said of Genzyme’s new supply disruption. “I’m certain it will have some impact on patients. It’s extremely unfortunate that this has occurred, and very frustrating. But they’ve made it clear disruptions could happen.’’
It was the second production setback for Genzyme in the past week. On Friday, the company said it expects a shortage of its thyroid medicine, Thyrogen, from mid-April to mid-June because of a separate manufacturing problem.
Genzyme, which had to temporarily shut down the Allston Landing plant in 2009 and ration both Cerezyme and Fabrazyme, is completing a two-year manufacturing recovery program. Even with the fill-finishing disruption, Gorski said, the company remains on track to return to full Fabrazyme supply by the second half of this year. It already has resumed full shipments of Cerezyme, which treats patients with Gaucher disease. Both Gaucher and Fabry diseases are caused by enzyme deficiencies.
The company in February reached an agreement to be acquired for $20.1 billion by French pharmaceutical giant Sanofi-Aventis SA, a deal set to be completed next month. Sanofi has said it plans to use Genzyme as its global hub for drugs to treat rare diseases.
“Sanofi is certainly aware of this disruption, but they’re not actively involved in our operations at this time,’’ Gorski said.
Under the sales deal, Genzyme investors will receive milestone payments, called contingent value rights, if its drugs meet production or sales targets, including a 2011 production target for Fabrazyme. Gorski said Genzyme expects to meet the milestone despite the setback.
In its notice to Fabrazyme patients yesterday, Genzyme said the timing and extent of reductions in the drug’s availability in coming months would vary from region to region around the globe. “All regions will share the impact of the reduction in supply,’’ the company said.
A spokesman at Sanofi’s US headquarters in Bridgewater, N.J., had no immediate comment on the Genzyme supply disruption. The parties have said they expect to complete the acquisition in the early part of the second quarter.
Robert Weisman can be reached at email@example.com.