Biogen Idec to seek stronger warning label for MS drug, CEO says
By Mark Jewell, AP Business Writer August 31, 2005
CAMBRIDGE, Mass. --The head of Biogen Idec Inc. said in an interview Wednesday his company will recommend to regulators within a month that the warning label be strengthened on a multiple sclerosis drug the company hopes to return to the market despite safety concerns.
James Mullen said Biogen Idec will likely recommend that Tysabri include warnings about three cases of an often-fatal brain disease that were confirmed after clinical trials of the drug, which was withdrawn from the market Feb. 28 despite hopes that it would become an important new tool in treating MS.
Mullen said the revised label that the company will propose to the U.S. Food and Drug Administration also will warn about risks for patients who have weak immune systems and therefore could be more susceptible to contracting the disease, called progressive multifocal leukoencephalopathy, or PML.
However, Mullen said the label language his company will suggest to FDA when it seeks permission to resume marketing Tysabri will acknowledge that scientists don't yet understand precisely how the bioengineered drug put the three patients who contracted PML at risk of contracting the rare disease. Two of those patients died.
"I think it's important for us to not overstate what we know," Mullen, Biogen Idec's chief executive and president, said in an interview with The Associated Press at the company's Cambridge headquarters. "We want to be concrete about what we know and what we don't know."
Mullen said Biogen Idec and its Irish partner in Tysabri, Elan Corp., plan to submit findings from their review of the drug's safety to the FDA by the end of September.
The companies had previously said the report would be submitted sometime in the fall so the FDA could review whether Tysabri can safely return to the market. Some industry analysts have said strong warnings on Tysabri's label could ruin the drug's chances of becoming a commercial success.
Mullen said it is "highly unlikely" the FDA will complete its own review by the year's end, but he said the regulators are "interested in a thorough but expeditious review of determining whether this can brought back to the market in the near future or not."
FDA spokeswoman Lenore Gelb declined to comment Wednesday.
Biogen Idec and Elan said Aug. 9 that more than 2,000 patients with multiple sclerosis who took Tysabri in clinical trials had been screened for PML as part of the companies' safety review, but no new cases were found.
The companies are close to finishing a similar review of about 1,500 people who took Tysabri in clinical trials to test its effectiveness in treating Crohn's disease and rheumatoid arthritis.
After reviewing one year of data from planned two-year trials, federal regulators in November approved Tysabri for sale to the 350,000 American sufferers of MS, a debilitating and incurable disease in which the body's immune system turns rebellious, attacking, inflaming and damaging its own nerve tissue.
The drug was withdrawn about three months after its approval, causing Biogen shares to plunge more than 42 percent the day of the announcement while Elan's stock fell 70 percent.
"We hope there is a pathway back to the market," Mullen said. "We think that unless we see some new surprises, that the risk-benefit profile for this product certainly warrants it being used for MS.
"We've got probably hundreds of letters from patients saying, 'Tysabri changed my life. I've got no other options. I'll sign whatever waiver I need to get access to this product.'"
MS drug's predicted sales are lowered
By Jeffrey Krasner, Globe Staff | August 30, 2005
Even if Biogen Idec Inc. is able to win regulatory approval to sell its multiple sclerosis drug Tysabri, sales probably will not be enough to have a significant impact on the Cambridge company, according to a report by a Wall Street analyst.
Steven Harr of Morgan Stanley said that instead of the $3 billion in annual sales once predicted by some analysts, Tysabri would likely become a ''niche" drug with yearly sales of about $500 million.
Harr also highlighted a broad range of complications experienced by multiple sclerosis patients taking the drug during clinical trials. He said those adverse events, including unusual infections, will play a role when Biogen Idec and Elan Corp. of Ireland, its partner in Tysabri, meet this year with Food and Drug Administration officials to discuss reintroducing the drug.
''Ultimately, we believe there is a reasonable chance this drug returns to the market, but its commercial impact may be limited," Harr wrote to clients on Friday. Patients taking Tysabri may have to be closely monitored for signs of opportunistic infections, he said.
Biogen Idec and Elan voluntarily withdrew Tysabri from the market in February, three months after it went on sale, after learning that a woman taking the drug in a clinical trial had died of PML, a rare brain disease. A second patient was later diagnosed with PML and survived, and a third patient involved in a separate trial was later diagnosed as having died of the disease two years earlier.
In a call to investors this month, Biogen Idec officials said they hadn't found any additional cases of PML, or progressive multifocal leukoencephalopathy. The company also looked at infections among multiple sclerosis patients in Tysabri trials and found no significant variations between those taking the drug and those receiving a placebo.
''We looked at any kind of atypical or opportunistic infection," said Amy Brockelman, a Biogen Idec spokeswoman. ''We saw a range including herpes simplex encephalitis, cancer, vascular events, urinary tract infections, and pneumonia. The end result was we found no safety signals, infectious or otherwise."
The infections Harr referred to in his report were broadly discussed during the conference call to investors, she said.
Biogen Idec is continuing its safety review of Tysabri by looking at patients who took the drug in trials for Crohn's disease and rheumatoid arthritis. It says it will submit a plan to the FDA this fall for returning the drug to the market, including a suggestion for a revised warning label for Tysabri.
In his report, Harr said he saw little reason for Biogen Idec shares to appreciate beyond his target of $42 without an additional catalyst such as a blockbuster drug. Shares closed yesterday at $40.99, down 6 cents. Dr. Douglas R. Jeffery, a neurologist at Wake Forest University in Winston-Salem, N.C., said the additional infections highlighted in the report will have to be evaluated by the FDA.
''The question is, were there enough of these infections to be statistically significant, and did they occur more often in the Tysabri-treated group than the general population?" Jeffery asked.