Users ask FDA panel for return of Tysabri
GAITHERSBURG, Md. -- Dozens of multiple sclerosis patients told a Food and Drug Administration advisory panel yesterday that they should be allowed to take Tysabri, a promising treatment for MS pulled from the market last year after it was linked to a rare but deadly brain disease.
Biogen Idec of Cambridge and its Irish partner Elan Corp. PLC, the drug's makers, said they have developed a risk management plan that could allow Tysabri's return as a treatment for a small group of patients.
The advisory panel will vote on Tysabri's fate at the conclusion of today's session. The FDA often follows its advisers' guidance.
Yesterday, advisers heard often heart-rending testimony from MS patients and their families.
Heather Smith, 36, said Tysabri gave her more energy and allowed her to move about without a wheelchair, which she is now forced to use again. Smith sobbed so frequently while describing how her symptoms worsened since Tysabri was withdrawn that she was cut off before she finished.
Another patient, Pamela Sue Clark, 41, testified that the drug helped her feel well enough to walk to a pond near her home with her 5-year-old sons.
''And I did smile more often," Clark said. ''That is what hope does."
Advisers were also scheduled to hear from Walter Smith, who is suing Biogen Idec because his wife, Anita, 46, died from the brain disease, progressive multifocal leukoencephalopathy, after taking Tysabri in a clinical trial.
Walter Smith, however, was too overcome with emotion to deliver his statement. Instead, the couple's daughter, Beth Smith, spoke and said the family was ''never told" Tysabri could lead to her mother's death.
Gregory Shoukimas, a doctor who reviewed Anita Smith's medical records after her death, said she did not meet the definition of an MS patient. The decision to include her in a trial for a drug that led to her death raises ''serious concerns that Biogen Idec is incapable of proceeding in a safe manner with future clinical trials," he said.
Tim Hunt, a Biogen Idec spokesman, declined to comment. But the advisory panel still peppered company representatives with questions about whether Anita Smith had MS.
''I did not see the patient," said Dr. Alfred Sandrock, Biogen Idec's vice president of neurology. ''I don't like to second guess my colleagues."
Biogen Idec told the panel it can reduce risks of PML by ensuring patients are carefully selected, fully informed of risks, and closely monitored for neurological changes. It also volunteered to add a black box -- the FDA's most serious warning -- to the Tysabri label, and would instruct doctors not to give the drug to patients with compromised immune systems.
Under the company's plan, doctors who prescribe Tysabri and patients who receive it would participate in a mandatory registry to ensure the right patients get it and know its risks. Patients could only get monthly infusions at registered centers.
The stakes for Biogen Idec and Elan, and for competitors offering rival drugs, were high enough to draw a standing-room-only audience in the hotel ballroom where the two-day hearing is being held.
One MS patient arrived in a cherry red wheelchair sporting yellow fuzzy dice. Another's wheelchair was emblazoned with ''Got Tysabri?" in glitter.
Although it typically approves such drugs after two-year trials, the FDA gave speedy approval to Tysabri in November 2004 based on one year's data, because its efficacy was ''so robust." The drug was taken off the market voluntarily by the companies in February 2005 after they learned of two cases of PML. The company later confirmed a third case. Two of the three patients died. Until then, the drug was expected to attain blockbuster status potentially reaching up to $3 billion in annual sales.
A safety review by the companies did not uncover additional PML cases, and last month the FDA approved Tysabri for patients in a clinical trial.
MS, an incurable disease that predominantly affects young women, turns the body's immune system against tissues that insulate nerves. It strikes without notice and inevitably leads to disabilities.
Within 15 years, MS patients often need a cane or crutch to walk, and many are wheelchair-bound within 25 years. While not life threatening, MS reduces life expectancy by up to seven years.
Patients taking Tysabri may experience a two-thirds reduction in MS flare-ups, about double the benefits offered by other MS drugs.
Dr. Robert Temple, who oversees the FDA division that handles neurology drugs, stood by the decision to stop sales of Tysabri.
Even if it is allowed to return to the market, he said, the drug will be closely scrutinized for PML risks.
Diedtra Henderson can be reached via e-mail at dhenderson@globe.com. 