Crisis Averted, But No Long-Term Fix

March 20, 2012
Amy Ritter, PhD

Amy Ritter was Scientific Editor, BioPharm International.

A critical drug that has experienced shortages is Genzyme’s, Fabrazyme (agalsidase beta), the only enzyme replacement therapy approved in the US for Fabry disease.

A critical drug that has experienced shortages is Genzyme’s, Fabrazyme (agalsidase beta), the only enzyme replacement therapy approved in the US for Fabry disease. The drug was originally produced at the company’s Allston, Massachusetts plant. The Allston plant was plagued with quality problems, which resulted in a consent decree, temporary closure of the plant in 2010, and rejection of some lots of Fabrazyme for quality reasons. In January of 2012, FDA granted approval for Genzyme to start producing Fabrazyme at its Framingham, Massachusetts plant, bringing to an end a shortage that left patients without an adequate supply of medication for nearly two years.

While Fabrazyme was in such short supply, some US patients were allowed limited access to Replagal (agalsidase alfa), a biologic produced by Shire Pharmaceuticals and approved in Europe for the treatment of Fabry disease. During the Fabrazyme shortage, FDA encouraged Shire to seek US approval of Replagal, which would have meant that there would have been a readily available alternative treatment for Fabry patients.

Shire announced on Mar. 14, 2012 that it was withdrawing its Biological License Agreement (BLA) for Replagal. In its press announcement, Shire says, “Recent interactions with the FDA have led the Company to believe that the agency will require additional controlled trials for approval. No concerns over the product’s safety profile were raised by the FDA. Shire has concluded that the likely additional studies would cause a significant delay, and an approval of REPLAGAL for US patients would only be possible in the distant future. Shire has therefore decided to withdraw its BLA.”

Apparently, Shire decided that a speedy approval was not forthcoming, and decided to take its ball and go home. Perhaps it did the calculation, and decided that the market share it might pick up in the US didn’t justify the cost of the additional trials. Whatever the reason, the ones affected are the patients, who are still depending on a single supplier to keep them healthy.

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