MS sucks. I'm not minimizing the difficulty of developing effective treatments for this disease. I just think it would be good if patients had full information on the brain infection that might kill them as a result of one treatment option.

 

 

BUSINESS

OCTOBER 26, 2009

Europe Takes Closer Look At Tysabri

 

By THOMAS GRYTA

A European panel has started a review of the controversial multiple sclerosis drug Tysabri, sold by Biogen Idec Inc. and Elan PLC, citing a higher rate of a rare brain infection than previously disclosed.

The additional cases are important because they may increase the drug's risk profile and raise questions about the companies' responsibility in updating the market on the safety record of Tysabri, a product that generates nearly $1 billion in yearly revenue.

The European Medicines Agency's Committee for Medicinal Products for Human Use, known as CHMP, reported there have been 23 cases of progressive multifocal leukoencephalopathy, or PML, a number that the Food and Drug Administration later confirmed, adding that those cases were after Tysabri returned to the market in 2006. The FDA said it is continuing to assess the situation.

Previously, there had only been 13 confirmed cases of the infection since the drug re-entered the market in 2006, after being removed for 18 months because of a link to three other PML cases.

Tysabri was allowed back on the market after patients and physicians pushed for its return. The drug, despite the PML risk, is seen as one of the most effective MS treatments on the market, especially for those with severe cases who have few other options.

The European panel said its review will discuss "any additional measures necessary to ensure the safe use of Tysabri and how to balance the risks to the patients against the benefits of the treatment."

Christopher Raymond, an analyst with Robert Baird, said European regulators are unlikely to remove it from the market, but they may recommend that long-term patients take a break from using the drug to help balance risk. Duration of use is seen as raising the risk factor for PML.

Earlier this week, Biogen acknowledged that it is talks with the U.S. Food and Drug Administration to amend Tysabri's label to reflect increased PML risk with longer-term usage. Changing the label marks a shift for Biogen, which has long maintained there was no clear connection to duration and increased risk of PML.

The increased potential of patients taking a break, or "drug holiday," from Tysabri could affect its sales growth, and that prospect weighed on shares of Biogen and Elan on Friday. Shares of Cambridge, Mass.-based Biogen fell 5.8% to $44.49, while the American depository receipts of Dublin-based Elan plunged 20% to $5.17 in Friday afternoon trading.

Biogen spokeswoman Naomi Aoki said the incidents of PML remain rare, reiterating the company's contention that the rate remains within the 1-in-1,000 patient level implied by its label.

As of Sept. 30, about 46,200 people world-wide were taking Tysabri with about 13,400 patients on the drug for more than 24 months. Because duration is seen as an issue, there is a debate as to which number to compare to the number of PML cases—either the total number of patients or those on Tysabri for two or more years.

Ms. Aoki stressed the strong effectiveness of the drug in treating the debilitating disease and said the risk-benefit profile remains favorable. Ms. Aoki, citing company policy, declined to comment on the number of PML cases reported by the European agency.

"We don't think it is beneficial to comment on the numbers because it is within the [label's] rate," she said.

Officials from Elan weren't immediately available to comment.

Sanford Bernstein analyst Geoffrey Porges said the significance of the cases is hard to gauge without further details, but valued Biogen's stock at $41 without Tysabri.

"These new cases are likely to alarm physicians whose comfort with the product had been increasing in recent months, and should catalyze academic and regulatory discussions about more active risk mitigation strategies," he wrote in a note to clients.

The new cases also might reignite a debate over disclosure of new PML cases. Neither company regularly provides such updates, which has caused some frustration on Wall Street.

Biogen was giving weekly updates until July 24, the third anniversary of Tysabri's relaunch, but now maintains that disclosing such information isn't helpful because the PML rate is already on the label and FDA is aware of all new cases. Thus, the new information only creates fear among patients and physicians.

Furthermore, Biogen has said, such negative information can be used by other drug makers in the increasingly competitive MS treatment market.

Write to Thomas Gryta at thomas.gryta@dowjones.com