UPDATE: Genzyme: US Cerezyme Dosing Returns To Normal In Sept

Genzyme Corp. (GENZ) will return U.S. patients using its top-selling Cerezyme to normal dosing in September, as well as increase dosing for Fabrazyme patients, both moves coming a month earlier than expected amid a long shortage of the rare disease treatments.

Patients using Cerezyme, used to treat the rare genetic disorder Gaucher's disease, and Fabry disease drug Fabrazyme, generally haven't been able to use the drugs normally since last year because of problems at an Allston, Mass., production facility, the sole source of the products.

Patients affected by both diseases lack specific enzymes - which the drugs replace - that break down certain types of fats, causing them to build up in the body and leading to major problems including pain, skeletal complications, organ failure and death.

In the U.S., Cerezyme patients will be returning to normal dosing in September, and the company will start adding new patients from a waiting list during the month. That list includes both patients that have stopped using the treatment, and those that haven't been treated before.

Across the world, patient doses of the drug will increase in September in most markets and will return to normal in the fourth quarter, the company said.

Although it is confident in continuing the supply of Cerezyme, Genzyme will update its supply plan again in late September.

For Fabrazyme, production has been plagued by efficiency problems since the restart of the Allston plant, but the company said U.S. patients will also increase dosing in September followed by the rest of the world in the fourth quarter.

"We are not as positive as we are about Cerezyme, but we are continuing to make progress," said John Butler, president of Genzyme's personalized genetic health business on Thursday.

Fabrazyme patients in the U.S. will receive a dose a month for September and October. They had received a dose every other month for the past few months, the company said.

For both drugs, normal dosing generally means getting the drug twice a month. Many Cerezyme patients have been getting the drug once a month during the shortage. Genzyme continues to have no projection for when Fabrazyme patient can return to normal dosing.

Genzyme had previously said it would return Cerezyme patients to normal dosing and increase dosing in Fabrazyme in the fourth quarter.

The news comes as Genzyme has been under siege from activist investors in recent months, and is reportedly in takeover talks with France's Sanofi-Aventis SA (SNY).

The Allston plant has been plagued by problems, including numerous failed regulatory inspections and a temporary shutdown last year due to contamination, something that has caused product shortages and hurt Genzyme's earnings. The company recently extended the timeline for fixing the issues by a year, meaning regulatory oversight of the plant could last as many as nine years.

Cerezyme sales dropped 34% last year to $793 million due to the shortage, while Fabrazyme sales dropped 13% to $494 million. The company had total revenue of $4.5 billion.

The problems have allowed the entrance of competitors to the drugs. Shire PLC (SHPGY SHP.LN) makes Gaucher's treatment Vpriv, which was approved in the U.S. in February and in Europe on Thursday. It already sells Fabry disease drug Replagal in Europe and plans to file for U.S. approval.

Protalix BioTherapeutics Inc. (PLX) is developing a Gaucher's disease treatment with Pfizer Inc. (PFE) that is under Food and Drug Administration review.

Because of the shortages, the FDA allowed all the drugs to be used under a special protocol last year, despite not being approved for marketing.

Although Genzyme's Allston facility is fully operational for production purposes, Butler expressed confidence in maintaining patients on the increased dosing, but warned the situation remains fragile.

"We are working without inventory, so any minor disruption could cause an issue for us," Butler said.

Shares of Genzyme recently traded down 50 cents to $67.08.

-By Thomas Gryta, Dow Jones Newswires; 212-416-2169; thomas.gryta@dowjones.com

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