Genzyme dominates the market for treating Gaucher’s disease, each year selling more than $1.2 billion worth of its drug imiglucerase (Cerezyme) to patients with the rare genetic disease, who need to take the treatment throughout their lives. But competitors are flocking in to grab some of the market now that the Cambridge, MA-based biotech is running low on supplies. Switzerland-based Actelion, the maker of the only other FDA-approved treatment for Gaucher’s, is one of the companies hoping to fill the void.
Genzyme (NASDAQ: [[ticker:GENZ]]) has had to answer a lot of questions from doctors, patients, and investors since June 16, when it said it discovered a viral contamination at its Allston, MA-based manufacturing plant, where it makes imiglucerase and other therapies for rare genetic disorders. The contamination meant the Genzyme plant would need to be shut down and disinfected, causing a supply interruption which the company now estimates will last six to eight weeks, beginning in August.
This situation has created plenty of consternation for patients with Gaucher’s (go-SHAYZ). They need to stay on their medication to prevent the buildup of a fatty substance in their spleen, liver, lungs, and bones—which can keep those organs from functioning properly and lead to extreme fatigue, and even death. The Genzyme drug, approved by the FDA back in 1994, is an enzyme replacement therapy that is generally “very beneficial” for patients with the most common form of Gaucher’s, according to the National Institutes of Health.
Actelion’s drug miglustat (Zavesca) came along in 2003, when Genzyme had already secured a dominant position in the market, and is still a small player in the treatment of Gaucher’s. Only about 90 patients in the U.S., and 300 total worldwide, currently take the Actelion drug, generating about $36 million in revenue last year, says Kirk Taylor, the senior vice president of Actelion’s U.S. medical department. So Actelion’s task will be to persuade doctors and patients to give miglustat a try during the shortage
