Genzyme patients disillusioned by production snafus

Genzyme has been fighting many fires over the past year: Plant contamination, a potential FDA consent decree, a proxy fight with Carl Icahn, to name a few. Its manufacturing troubles have led to shortages of its drugs for genetic disorders, but we haven't witnessed how those shortages have been affecting patients.

Now we can. The New York Times looks at patients who've been using Genzyme's drugs, and finds that some are disillusioned about a company they once considered a lifesaver. Lack of their medicines has put patients in pain--and may have contributed to at least one death.

But because there are few--if any--alternatives to Genzyme's treatments, those patients can't just walk away because they feel betrayed. "Unfortunately, they have the drug we need," explains one patient.

The patients question why Genzyme didn't have a larger reserve of its products for Gaucher disease and Fabry disease, given that they're so crucial to the well-being those patients. CEO Henri Termeer tells the NYT that reserves were low because Genzyme had temporarily been using its Boston facility to produce a drug for another rare disorder, Pompe disease. He also admits that his company has work to do before patients can trust it again. "We have to re-earn our standing with these patients," he says.

Because of the shortages, some potential competitors for Gaucher treatment Cerezyme took big leaps forward. To help mitigate those shortages, FDA let doctors prescribe not-yet-approved treatments from Shire and Protalix, giving those drugs a foothold in Genzyme's territory. Will disappointed patients be eager to turn to them when they're on the market for real?

- see the NYT article