CAMBRIDGE — Is this city big enough for two multiple sclerosis drug makers?
We're about to find out.
With Genzyme’s disclosure Monday that the Food and Drug Administration accepted for review its revised application to sell the experimental drug Lemtrada in the United States, the biotechnology company moved a step closer to building a multidrug MS franchise based in Kendall Square — blocks from the research campus of the world’s leading maker of MS treatments, Biogen Idec Inc.
“There’s still a tremendous unmet need for better drugs, more effective drugs, and more convenient drugs,” said Bill Sibold, senior vice president and head of the MS business at Genzyme, which last fall won FDA approval of its first MS therapy, Aubagio.
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. Genzyme’s push into the lucrative market, which analysts estimate at $13 billion annually, is a departure for the biotech pioneer known for developing drugs to treat rare genetic disorders such as Gaucher and Fabry diseases, which affect smaller numbers of patients.
It hasn’t escaped notice locally that Genzyme’s new business is taking shape in the shadow of Biogen Idec, which already produces two best-selling MS treatments and is actively working to develop a broader MS portfolio. Genzyme’s entry into the field underscores the region’s growing prominence as a center for cutting-edge drug development, analysts said.
“When it comes to MS or neuroscience in general, it’s all about attracting talent and critical mass,” said Kevin J. Gorman, managing partner for Putnam Associates, a life sciences consulting firm in Burlington. “Biopharmaceutical companies want to be in the Boston area, and if you take it down to a specific therapeutic area, the same rule holds.”
Biogen Idec is awaiting an FDA decision in March on its application for approval of a pill now called BG-12 that would be its first oral treatment for MS. And last week, the Weston-based company reported positive results in late-stage clinic trials for its peginterferon drug candidate, which would offer patients prolonged exposure to the medicine so they wouldn’t have to be injected as often as similar treatments that are now available.
“It’s a very exciting time for Boston,” said Gilmore O’Neill, vice president of global multiple sclerosis clinical development at Biogen Idec. “You’ve got a significant cluster of MS drug activity in the Boston area. Genzyme’s a potential competitor, but these drugs all have different mechanisms. It’s a matter of getting the right drug to the right patient.”
Lemtrada, the Genzyme experimental drug, figured prominently in the company’s protracted negotiations with the French pharmaceutical giant Sanofi AG, which ultimately bought the Cambridge company for $20.1 billion in 2011. Unable to agree on the drug’s potential market value, the two companies agreed to offer Genzyme shareholders so-called contingent value rights. Under that arrangement, stock owners could get milestone payments tied to how well Lemtrada sells.
Genzyme has high hopes for Lemtrada in the “high efficacy” portion of the MS market, where it would compete with Biogen Idec’s drug Tysabri, among others, in treating a population that includes patients with more severe forms of the disease. But after originally submitting its application for FDA approval last June, Genzyme suffered a setback in August when the agency asked the company to change the way it presented clinical data and resubmit the application.
Now that the FDA has accepted the revamped application, “we’re very excited that the [review] process has begun,” said Michael Panzara, a former Massachusetts General Hospital neurologist who is therapeutic area head for Genzyme’s MS and neurological research program. Genzyme anticipates decisions by US and European regulators later this year that would enable it to start selling Lemtrada on both sides of the Atlantic.
After it acquired Genzyme two years ago, Sanofi transferred its own MS drug candidate, Aubagio, from its Bridgewater, N.J., labs to the Genzyme research center in Framingham where it was folded into Genzyme’s MS program.
“If you look at the dynamics in the MS space, either company by itself would just be another company with a product,” said Sibold, who like Panzara is a veteran of the MS business at Biogen Idec. “To me, having a franchise is really important.”
Genzyme’s two drugs are aimed at different segments of the market and have significantly different dosing regimens. While Aubagio is a pill, Lemtrada is administered intravenously for five days the first year of use, and for three days the next year. Genzyme began selling Aubagio last October, but has yet to report sales figures.
Biogen Idec, similarly, has tried to differentiate its MS drug offerings. Its original drug, Avonex, was one of the first interferons, a class of protein-based drugs sometimes called “platform treatments” for the disease. Sales of Avonex climbed 8 percent to $2.9 billion last year, according to financial results posted Monday by Biogen Idec.
Tysabri was temporarily pulled from the market in 2005 after some patients died from a rare brain infection. But Biogen Idec has since developed a test to predict which patients might be vulnerable to the infection. Tysabri sales increased 5 percent to $1.1 billion last year.
Securities analysts don’t believe Genzyme will challenge Biogen Idec’s dominance in the overall MS market in the near future. But Lemtrada “might pressure Tysabri on the margin in the “high efficacy segment,” said Mark Schoenebaum, biotechnology analyst for investment strategy firm ISI Group in New York.
At least two other companies with large operations in Massachusetts are also competing in the MS market. German-owned EMD Serono of Rockland sells Rebif, another interferon. And Swiss drug giant Novartis AG, which has its global research center in Cambridge, developed the first oral MS treatment, Gilenya, which will compete with Aubagio and the Biogen Idec pill. But much of the research and development of those therapies was done elsewhere.
Here in Massachusetts, Genzyme hopes to some day join Biogen Idec as a leader in MS treatments.
“If I didn’t believe there was the potential for Genzyme to build a diverse portfolio and really attack the disease in a special way, I wouldn’t be here,” Panzara said.