Pill for Gaucher disease helps strengthen bone

Genzyme Corp said on Thursday its experimental pill to treat Gaucher disease was effective in several ways. It strengthened bone, increased blood count, and decreased the size of bloated organs by a significant amount.

Results from the two-year trial were presented at the Lysosomal Disease Network World Symposium in Miami and suggest the drug may be at least as effective as the company’s best-selling Gaucher disease drug, Cerezyme.

“This is not a head-to-head comparison, but based on the historical experience we’ve seen with Cerezyme the results look comparable,” Genzyme Senior Vice President Geoff McDonough said in an interview.

Gaucher disease is a rare genetic disorder in which patients are deficient in an enzyme that breaks down certain fats in the body, leading to swollen, damaged organs and even death.

Genzyme said its new drug, known as eliglustat tartrate (formerly GENZ-112638), may be more effective than Cerezyme in improving bone density. Bones often become weak and brittle in patients with Gaucher disease.

“One of the major medical needs that is not met well in Gaucher is treatment of the bone,” McDonough said. “What we’re seeing today suggests to us that we may have a more effective treatment in the bone department than we have seen historically with enzyme replacement therapy.”

Data showed that after two years of treatment with the new drug, patients whose bone mineral density had been below the normal range at baseline began approaching the normal range.

“We normally start to see these changes with enzyme replacement therapy after four to five years,” McDonough said.

Cerezyme, like rival products under development from British drugmaker Shire Plc and Protalix Biotherapeutics Inc of Israel, is given by infusion. A Cerezyme-like product that could be given in capsule form would be far more convenient for patients and could potentially give Genzyme a competitive advantage.

The Cambridge, Massachusetts-based biotech company is struggling to rebuild its reputation with patients and with Wall Street after manufacturing problems last year lead to shortages of Cerezyme and Fabrazyme, its drug to treat Fabry disease.

The latest trial of eliglustat tartrate showed that patients who received the drug for two years had an average decrease in swollen spleen volume of 52 percent and an average decrease in swollen liver volume of 24 percent.

Hemoglobin levels increased from baseline by an average of 2.1 grams per deciliter, while platelet counts increased by an average of 81 percent.

By Toni Clarke

BOSTON (Reuters)