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Genzyme Rival, Actelion, Seeks to Fill Void Created by Cerezyme Shortage

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of the Genzyme drug, he says.

“I hope that doctors will put patients first and realize that patients need their medicine on a regular basis,” Taylor says.

Both drugs are expensive. The Genzyme drug costs $200,000 per patient each year, and the Actelion drug is priced at about $120,000 a year. But the drugs work quite differently, and require different habits among patients. Genzyme’s drug is given via infusion into a vein over a one to two hour period, as often as three times a week, or as infrequently as once every three weeks. The drug is a genetically engineered analog of an enzyme that is lacking in patients with Gaucher’s. Its job is to help break down the fatty substance that would otherwise accumulate in their organs and tissues.

The Actelion drug is an oral capsule that needs to be taken three times a day, Taylor says. Instead of replacing the deficient enzyme, it blocks the action of a different enzyme that is involved in making the fatty substance in the first place, he says. The drug is typically taken by patients who prefer an oral treatment, or don’t do well on enzyme replacement therapy, Taylor says.

Actelion hadn’t been contacted by the FDA when I interviewed Taylor on July 6, although the agency has reportedly made inquiries to Israel-based Protalix BioTherapeutics and U.K.-based Shire, which has operations in Cambridge, MA, and Lexington, MA, about potentially making their experimental drugs for Gaucher’s available to patients to help cope with the shutdown. It’s a fast-moving situation: Shire said on July 7 that it applied for FDA approval of its enzyme replacement therapy for Gaucher’s, at the FDA’s request.

Actelion isn’t saying how much market share it hopes to capture because of the Genzyme shortage, the company has seen “an uptick” in prescriptions since the shortage was first announced last month, Taylor says. The company has ample supplies, and its sales reps and medical science liaisons are ready to answer doctors’ questions about the safety and effectiveness of their drug, he says.

“This is a great opportunity for Actelion to show that for us it’s not all about blockbuster sales, but about helping patients,” he says.

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