Thursday, March 24, 2011

Genzyme hit with more Fabrazyme drug woes

By Lori Valigra, Mass High Tech correspondent

Genzyme Corp. said one lot of its Fabrazyme drug for Fabry disease, a rare genetic disorder, had to be scrapped in the final stages of production at its Allston plant. The Cambridge biotech said that it plans to be able to return to normal production in the second half of this year.

“Production of the bulk material to make the product is proceeding in Allston, and we are sending the fill and finishing operation to a third party,” spokeswoman Lori Gorski said. That third party company is Hospira, in Kansas.

“The issue was in our fill and finish area, where the drug is filled and capped and placed into vials. The monitoring conditions in that area of the plant were not met,” she said, adding that the area did not meet “current good manufacturing practice (CGMP),” an industry benchmark for quality assurance in manufacturing. She said the area where Fabrazyme is freeze-dried and placed into vials is continually monitored. She could not provide a date when the incident occurred nor would she disclose the amount of the drug affected.

Fabrazyme has been in short supply since the Allston plant was contaminated and subsequently shut down for a while in June 2009. Genzyme voluntarily recalled 21 lots of the drug, which translated into 18,295 vials of 5 mg Fabrazyme for intravenous infusion, after receiving a warning letter from the U.S. Food and Drug Administration (FDA). The company was fined $175 million last April by the FDA, which also slapped it with a consent decree.

Genzyme informed patients of the most recent incident on its supply update website, noting, “As you know, we have been operating with very limited inventory of Fabrazyme. Because inventory is so limited, loss of this specific lot of Fabrazyme will have an impact on some patients in the coming months. For some patients, this may mean delaying scheduled infusions immediately; for some patients it may mean missing one or more infusions altogether over the next few months. All regions will share the impact of this reduction in supply.”

The website also said, “We are still on track to return to normal supply of Fabrazyme in the second half of 2011 with the expected approval of our new manufacturing facility in Framingham, Ma. Until this new facility is approved, Fabrazyme inventory remains low and supply will be vulnerable to disruption due to unforeseen manufacturing events such as this one.”

Genzyme has a couple thousand patients on the drug worldwide, Gorski said.

She added that despite the disruption, Genzyme expects to be able to meet the production milestone defined in the Contingent Value Right (CVR) in its $20 billion sale to sanofi-aventis. The CVR could add a payment if Genzyme hits the target of 79,000 viles of Fabrazyme this year. Gorski said sanofi is aware of the current production problem.

She said most Fabrazyme patients are on a half dose, although there is an emergency supply for a full dose for the most severe patients. “Our focus is on returning Fabrazyme patients to their normal dose,” she said.

However, the supply shortage prompted a civil lawsuit to be filed by patients in early March in Pennsylvania against Genzyme and the Mount Sinai School of Medicine.

In addition, Genzyme may feel pressure from Shire plc’s presentation of data last week showing that Fabry disease patients and type 1 Gaucher disease patients can safely transition from Genzyme’s therapeutics to Shire’s treatments for the diseases. Gorski declined to comment on that development.

The filling problem news did not appear to affect Genzyme’s stock (Nasdaq: GENZ), which rose slightly to $75.85 at 1 p.m., from the previous close of $75.63.