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: 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 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.