BOSTON (CN) – Patients with a painful, potentially fatal disease claim Genzyme, which makes the only FDA-approved drug for Fabry disease, sold Fabrazyme vials contaminated with glass, rubber and steel, and rationed the drug so that patients receive only half the approved dosage, while newly diagnosed patients are denied it altogether.
The federal class action says the rationing stems from Genzyme’s unending production problems.
It claims that Genzyme unreasonably used a publicly funded invention by “restricting administration to below the FDA approved dose” and by denying new Fabry patients access to the drug.
The class also sued Mount Sinai Medical School, which developed Fabrazyme, an enzyme replacement therapy, with help from a grant from the National Institutes of Health.
The plaintiffs suffer from Fabry disease, a life-threatening genetic illness caused by an enzyme deficiency. Fabry can cause pain throughout the body, and without treatment often leads to premature death from complications such as renal disease, heart attack and stroke.
While there is no cure for Fabry, Fabrazyme has been able to treat Fabry patients effectively, the class says. It adds that Fabrazyme, which is distributed only by Genzyme in the United States, is the only enzyme replacement therapy for Fabry approved by the Food and Drug Administration.
But “sometime before June 2009, Genzyme decreased production of Fabrazyme as a result of a viral infection in their Allston Landing, Mass. manufacturing plant,” according to the complaint.
It continues: “Genzyme caused the viral infection of Fabrazyme by failing to clean and sterilize their bioreactors between production batches, and thus introduced the virus by cross-contamination.
“Specifically, Genzyme would use the same bioreactors to produce both Fabrazyme and a different biological drug, Cerezyme, which is used to treat another enzyme deficiency termed Gaucher disease.
“The Cerezyme production batches were initially contaminated with the non-human virus Vesivirus 2117.
“Genzyme then cross-contaminated Fabrazyme cultures by failing to properly clean and sterilize the bioreactors before switching them for Fabrazyme production.
“The Allston Landing facility was the subject of a FDA warning letter that followed an inspection in September and October of 2008. One of the FDA’s concerns was controls to protect against microbial contamination.
“Further, in November 2009, Genzyme produced Fabrazyme vials that contained contaminants of particulate steel, glass and rubber.
“The FDA initiated action against Genzyme which resulted in a consent decree in May 2010, which included a $175 million dollar fine and oversight of the manufacture of Fabrazyme for at least 7 years.”
The production problems caused Genzyme to start rationing the drug in June 2009, the class claims.
“In June 2009, as a direct result of its reduced production of Fabrazyme, Genzyme unilaterally implemented a rationing plan for its reduced supply of Fabrazyme for the then known Fabry patients, wherein Genzyme unilaterally limited then known Fabry patients to receiving only less than one-third (1/3) of the recommended prescribed dose.”
Five of the named plaintiffs say they were receiving the recommended dose of Fabrazyme until then, “but after June 2009, Genzyme reduced their respective doses to less than one-third of the FDA approved dose pursuant to the Genzyme Rationing Plan.”
They add that “By and through the Genzyme Rationing Plan Genzyme also unilaterally barred any newly diagnosed patients from receiving Fabrazyme,” even though Fabry patients require immediate treatment.
Around January 2010, Genzyme “slightly increased doses to only 50 percent of the recommended prescribed dose,” and five named plaintiffs say they got that dose.
The class claims that Mt. Sinai knew of Genzyme’s rationing plan and “consented to Genzyme’s banning the drug to new patients despite having a duty to protect against the invention’s unreasonable use and non-use under the Bayh-Dole Act.”
The patients say that neither Genzyme nor Mt. Sinai applied for FDA approval of the rationing plan. And they say Genzyme did not test whether the reduced dose is safe or effective, and ignored the adverse effects that could result from the rationing plan.
A 2010 study by the European Medical Agency (EMA) found that the lower dosage caused more strokes, renal disorders and heart attacks in Fabry patients, according to the complaint.
The class says EMA’s study “showed that patients not only had a return of life-threatening symptoms but also an accelerated course of deterioration on the lowered dose.”
They say Genzyme failed to disclose the EMA study results, and that several Fabry patients died as a result of the shortage.
The class failed to persuade the NIH to override the Fabrazyme patent and to let other companies make the drug, the complaint states.
They seek compensatory and punitive damages for negligence, product liability, breach of warranty, deceptive trade practices and loss of consortium.
They are represented by Matthew Kurzweg of Pittsburgh.