Three patients taking Genzyme Corp.’s drug for Fabry disease, Fabrazyme, have petitioned the U.S. Department of Health and Human Services to use a rarely-used legal maneuver to open up the drug to competition.
On Monday, Joseph M. Carik, Anita Hochendoner and Anita Bova asked the federal government to invoke the Bayh-Dole Act’s march-in rights and grant an open license for other companies to use patents related to Fabrazyme. The law is designed to protect patients when a manufacturer with a monopoly on a drug cannot serve demand. There has been a shortage of Fabrazyme since the temporary closure of Genzyme’s Allston plant last summer following a viral contamination.
In their petition, the three patients allege that Genzyme has harmed the public health by severely rationing the supply of agalsidase beta, the only approved therapeutic treatment for Fabry disease.
The petition reads, in part, describing the patients’ health status:
“Their symptoms have worsened, and they are at greater risk of morbidity and death due to complications from the disease because of the severe and ongoing restriction in the supply of Fabrazyme. Their position is identical to all Fabry patients because all patients are being rationed the drug by Genzyme.”
Currently, there are no other approved drugs to treat Fabry disease. Shire plc, whose Human Genetic Therapies division is headquartered in Lexington and Cambridge, has a drug, Replagal, which is approved in Europe and has been submitted for approval to the U.S. Food and Drug Administration. U.S. regulators have been making Replagal available, pre-approval, on an emergency basis.
Jerry Walter, president of the National Fabry Disease Foundation, said in an interview, “There was such a flood of patients onto the Shire drug, through the treatment protocol, that they eventually said, ‘We can’t take any more patients until we find out if the drug is going to be approved.’”
There is a third potential treatment option for Fabry patients, an oral medication candidate produced by New Jersey-based Amicus Therapeutics Inc. The company is conducting a Phase 3 trial for the potential drug, and is recruiting patients. Only patients with a certain gene mutation, and who have not been taking Genzyme’s or Shire’s drug for at least the past six months, are eligible for the trial. Walter said that when the shortage hit, some patients stopped taking reduced doses of Fabrazyme to join the Amicus study.