By James DeGiulio —

In December 2009, the Department of Health and Human Services denied a petition filed by U.S. Fabry patients to secure higher quantities of drug Fabrazyme, exercise Bayh-Dole march-in rights against Genzyme, and issue an open license to U.S. patents needed to manufacture the drug (see "HHS Denies Request to Exercise Bayh-Dole "March-in" Rights for Fabrazyme").  Instead, the FDA entered a consent decree with Genzyme, which gave the FDA authority to regulate distribution of drugs during a shortage, which is the current status of Fabrazyme.  Still faced with the prospect of inadequate treatment options, according to a press release, U.S. Fabry disease patients have now petitioned the FDA to force Genzyme to keep more Fabrazyme for United States patients, rather than shipping it to Europe and other countries.  The initial production of Fabrazyme was sufficient to meet the needs of all patients in the United States.  However, in June 2009, Genzyme decreased production as a result of a viral infection at its manufacturing plant.  As a result of Genzyme's inability to meet the market need, doctors have been prescribing Fabrazyme in doses that are far below the indicated therapeutic use, current patients have been unable to obtain dosage increases, and newly diagnosed patients have been unable to receive therapy.

The petition filed with the FDA requests that Fabrazyme be allocated first to U.S. patients and, if it is not, that U.S. patients be allowed to present their case for medical need before the drug is sent overseas.  At present, only 38% of the total current drug production is actually allocated to U.S. citizens, with the majority of the drug being sold overseas, even though such patients have access to the alternative drug, Replagal, which is manufactured by Shire Pharmaceuticals.  There is no drug shortage in Europe, as the European Medicines Agency has recommended that patients either receive a full dose of Fabrazyme or be switched to Replagal.  The FDA has allowed some Fabry patients to import Replagal from Europe for emergency medical use in the U.S.  However, patients must petition the FDA and then show a critical medical need.

In the U.S., Replagal is not an FDA approved drug, and Shire has withdrawn its application for FDA approval, so no alternatives exist.  Citing the Bayh-Dole Act, the petition argues the public has a right of access to publicly funded inventions, and that depriving the public from this invention is a violation of Constitutional rights, specifically the Fifth Amendment.  According to the petition, by sending most of its Fabrazyme overseas, the current allocation system de facto denigrates the rights of U.S. citizens in favor of non-citizens.

In denying the petition to exercise Bayh-Dole march-in rights, the HHS relied on Genzyme reports indicating that they are very close to renewing and meeting the supply of Fabrazyme.  According to Genzyme, Fabrazyme engineering runs at the company's new Framingham manufacturing facility are nearly completed, and the material created there will become commercial product inventory upon regulatory approval of Fabrazyme production at this facility.  This approval is expected during the second half of this year and will enable the company to provide full, sustainable product supply.