In reversal, FDA approves Genzyme’s bid to sell MS drug in US

Eleven months after rejecting a powerful multiple sclerosis drug considered key to the future of Cambridge biotech Genzyme, federal regulators Friday reversed themselves and approved sale of the medicine, called Lemtrada.

The Food and Drug Administration decision came after MS patients across the country called for more treatment options and Genzyme submitted a new analysis of clinical data to address FDA concerns about how the company had designed trials to test the drug’s safety and effectiveness.

Patients have been anxiously awaiting the ruling on Lemtrada because studies showed people using it suffered fewer flareups of the potentially debilitating autoimmune disease, and about 70 percent of them were able to stop taking the injectable drug after two treatment courses.

“This is a great day for the MS community,” said Melissa J. Burdick, an MS patient from Waterford, Conn., who lodged one of several citizen petitions with the FDA after it refused to approve Lemtrada last December. “I’m absolutely thrilled beyond words to have this choice. Many people, including me, have wanted another treatment option.”

The company announced the FDA approval at about 9 p.m. Friday.

Genzyme executives said Lemtrada will be priced at $158,000 for two courses of treatment over two years. Rebif, a competing drug compared with Lemtrada in clinical studies, costs $134,600 for a similar treatment regimen, or 17 percent less. But the Genzyme executives pointed out that patients on Lemtrada suffered 50 percent fewer relapses than those taking the other drug.

Lemtrada’s approval is a vindication of French drug maker Sanofi SA’s move to buy Genzyme for $20.1 billion in 2011. Former Sanofi chief executive Christopher A. Viehbacher sealed the deal by creating a new stock that would reward Genzyme shareholders if the MS drug was approved by regulators and met sales milestones.

Viehbacher, however, did not last long enough to savor the success; last month he was fired by Sanofi’s board, which was critical of his management style. Despite his departure, the Paris-based company has said it remains committed to Genzyme and Lemtrada.

While the drug already has been approved in more than 40 countries — including European Union nations, Canada, Mexico, Australia, and South Korea — the United States is the world’s largest drug market.

“This is a very significant event for us,” said Bill Sibold, senior vice president and head of the MS business at Genzyme, which won FDA approval for its first MS therapy, Aubagio, in 2012. “About 60 percent of the global market for multiple sclerosis is in the US. We talk about our aspirations to be leaders in MS. Up until this point, we’ve had Aubagio, which we’re very proud of. But this will give us both products for our US franchise.”

Multiple sclerosis affects the brain and central nervous system of an estimated 400,000 people in the United States and 2.5 million globally. In the United States, the company said Friday, the use of Lemtrada would “generally be reserved” for patients who have previously taken two or more other MS drugs but are still having relapses.

Aubagio, a pill, was Genzyme’s first approved drug outside the field of enzyme replacement treatments for rare genetic disorders such as Gaucher and Fabry diseases. Sanofi recently reported worldwide sales for Aubagio totaled $378 million year for the first nine months of 2014, while revenue from Lemtrada, which has launched in only 10 countries, totaled $18 million.

But industry analysts have projected Lemtrada has the potential to become a “blockbuster” drug, generating annual revenue of more than $1 billion.

Unlike Aubagio, Lemtrada is administered at hospitals and clinics through infusions that take about four hours. Patients undergo the treatment for five consecutive days in the first course of therapy, and then for three consecutive days 12 months later. The drug showed “sustained efficacy” after three years for about 70 percent of patients, according to clinical studies, meaning they didn’t have take additional drug courses.

FDA regulators last year objected to Genzyme’s clinical trial design because patients knew whether they were taking Lemtrada or Rebif, a drug marketed by the Rockland biotech firm EMD Serono, but those assessing the trial data didn’t know. The regulators said they would have preferred a “double-blind” trial in which neither patients nor raters knew which drugs were being taken by individual patients.

Genzyme and patient advocates argued the side effects from the competing drugs were so distinct that it would be difficult, if not impossible, to conceal the identity of the treatments from patients in the trial. In resubmitting its application in May, the company provided additional materials sought by the FDA and a fresh analysis of the data to address concerns.

When it launches its second MS drug, Genzyme will boost its standing as an emerging rival to the leading MS drug maker, Biogen Idec Inc., which is based a few blocks from Genzyme in Cambridge’s Kendall Square.

Genzyme last week said it had begun enrolling patients in a clinical trial for a third MS drug candidate, a monoclonal antibody called VLA-2.

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