Shire’s Gaucher Drug Passes Key Trial, Putting More Heat on Genzyme During Shortage

8/3/09Follow @xconomy

Shire, the U.K.-based specialty pharmaceutical company, had some good news this morning for patients with Gaucher’s disease that might not be nearly as good for its competitor, Cambridge, MA-based Genzyme (NASDAQ: GENZ).

Shire said today that its experimental treatment for Gaucher’s, a rare genetic disorder, passed a key clinical trial in 25 patients. This is the first of three pivotal trials Shire is conducting, so this news might have gotten little notice except for one thing. The data is good enough that Shire persuaded the FDA to allow doctors to start prescribing the new treatment, velaglucerase alfa, before it’s ready for commercialization. Shire said it will provide free drug for patients who enroll in a special protocol.

This move clearly puts some additional heat on Genzyme, which is dealing with a shortage of its best-selling product, imiglucerase (Cerezyme), the market-leading enzyme replacement therapy for Gaucher’s. Genzyme’s Allston, MA-based manufacturing plant was plagued by a viral contamination in June, and the company shut the facility down for sanitizing, which created a shortage of Cerezyme production expected to last 6-8 weeks. This has created a serious concern for patients, who need to stay on steady medication to prevent the buildup of a fatty substance in their spleen, liver, lungs, and bones that can interfere with the function of those organs and lead to extreme fatigue, and death.

Other competitors besides Shire, like Switzerland-based Actelion, have sought to fill the void in the market during the Cerezyme shortage. Genzyme said in its second-quarter financial report that its sales of Cerezyme this year will likely be between $750 million to $1 billion, down from its previous forecast of $1.25 billion to $1.275 billion.

“With Allston supply issues front and center, it would be hard to imagine worse timing for Genzyme,” to face new competition from Shire, said analyst Christopher Raymond of Robert W. Baird & Co, in a note to clients today. This could erode Genzyme’s market share, especially if shortages continue, he said. “That said, we remain confident in Genzyme’s ability to bring Allston back up in a timely fashion.”

Genzyme shares fell 2 percent to $50.62 at 11:05 am Eastern.

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  • http://genzyme@hbsslaw.com Genzyme Investigation

    Hi All –

    Thought I’d provide a quick link for any investors reading this piece – I work for a law firm that’s investigating Genzyme on behalf of investors.

    You can learn more about the investigation here – http://www.hbsslaw.com/genzyme. HBSS is looking into possible securities violations around issues with plant inspections, FDA reports and how and when the company notified shareholders.

    Thanks!