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Patients, investors react to Genzyme's second attempt for approval of multiple sclerosis drug Lemtrada

Melissa Burdick
While yesterday’s news that Genzyme plans to resubmit an application for its previously-rejected multiple sclerosis drug couldn't have come too soon for patients, it came a little late for some investors.

Melissa Burdick, a 49-year-old patient from Waterford, Conn., said she was “thrilled” to hear the news. Burdick testified before the U.S. Food and Drug Administration in favor of the once-a-year drug, called Lemtrada, last year, and said in an interview Tuesday morning that she plans to do so again for the second application. Burdick filed a so-called “citizen’s petition” with the FDA less than two weeks after the Cambridge drug company’s Dec. 27 announcement that the agency turned down the application, Burdick appealed to the agency using what’s called a so-called “citizen’s petition” urging it to reconsider.
Burdick says that while the FDA is mandated to respond to such appeals, she has not received one yet and doesn’t know whether her appeal played a role in the agency’s reversal.

A main reason for the rejection, according to the company, was disagreement over the trial design. Namely, Genzyme said the rejection was partially due to the fact that the trials of 1,600 patients (which compared Lemtrada to EMD Serono’s drug, Rebif) blinded the doctors who rated the effectiveness of the drugs but not the patients themselves. When contacted in January, the FDA did not comment on its reasons for not approving a drug, but the FDA’s advisory committee also noted some adverse side effects, such as the fact that six patients on the drug developed thyroid cancers.

Story Source: The above story is based on materials provided by BIOFLASH
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