European regulators Tuesday put their stamp of approval on Genzyme’s multiple sclerosis drug Lemtrada, giving the Cambridge biotechnology company a second MS treatment for the overseas market only weeks after its first won approval there.
The move vindicated French drug maker Sanofi SA’s decision to pay $20.1 billion for Genzyme in 2011, a deal that hinged on the potential of Lemtrada, then an experimental therapy.
While it has not yet been approved for sale in the United States — the Food and Drug Administration is expected to rule on Genzyme’s application by the end of the year — Lemtrada won the European Commission’s blessing both for patients who have taken other MS drugs as well as newly diagnosed patients. It is expected to compete mainly against drugs such as Tysabri, sold by Cambridge-based Biogen Idec Inc., which is approved in Europe only for patients whose MS symptoms continued to progress even after they took other treatments.
European authorities on Aug. 30 approved Aubagio, an oral MS therapy developed by Sanofi but turned over to the Genzyme division. That pill was approved by the FDA for US sale last September, giving Genzyme its first entry into the MS market. The company is best known for developing drugs to treat rare genetic disorders such as Gaucher and Fabry diseases, which affect smaller numbers of patients than multiple sclerosis.
“We’re showing up in Europe to compete,” said Bill Sibold, senior vice president and head of the MS business at Genzyme. “Launching two products is certainly not without its challenges, but they’re challenges that anyone else in the industry would kill for.”
Multiple sclerosis, a potentially debilitating autoimmune disease, affects the brain and central nervous system of an estimated 400,000 people in the United States and 2.5 million globally. Securities analysts estimate the market for MS treatments at $13 billion annually.
Genzyme’s two drugs are aimed at different segments of the market and have significantly different dosing regimens. While Aubagio is taken orally, Lemtrada is administered intravenously for five days the first year of use, and for three days the next year. After that, many patients no longer need treatments for years, according to Genzyme’s clinical studies.
“What the data seems to suggest is we’re changing the course of the disease with this very focused treatment regimen,” Sibold said.
He said the drug targets and depletes cells that cause autoimmune damage, resulting in what he called “durable efficacy.”
Most prescription drugs in Europe have segmented labels indicating they are either for newly diagnosed patients or are to by taken by those for whom other treatments have failed. By contrast, Lemtrada’s label allows doctors to prescribe it for both groups of patients.
“The label we received is a change in the approach to MS in Europe,” said Michael Panzara, therapeutic area head for Genzyme’s MS and neurological research program. “It puts the treatment decisions in the hands of physicians and patients.”