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Waiting on Tysabri: MS patients line up as the drug returns to market(News & Observer, The (Raleigh, NC) (KRT) Via Thomson Dialog NewsEdge) Jul. 26--For a year, Lauren Hoffman has worn a red plastic bracelet stamped with the word "hope" wherever the three wheels of her walker would take her. The 57-year-old Cary woman is fighting a losing battle with multiple sclerosis. The bracelet represents the promise of a powerful drug, Tysabri. "It's the best thing that has come along for a long time," Hoffman said. "It keeps me from getting worse." Tysabri's makers, Biogen Idec and Elan, pulled the drug off the market in February 2005 after it was linked to the deaths of two MS patients. Both developed an otherwise rare brain infection, progressive multifocal leukoencephalopathy, or PML. Tysabri was projected to generate as much as $3 billion in annual sales. The recall was a huge disappointment for patients and physicians, as well as Biogen investors and employees, including 160 who were laid off at the company's Research Triangle Park operations. This week, the drug is returning to market after again winning regulatory approval in June. But its future is far from certain. This time, the drug carries tougher warnings, costs more and requires patients and doctors to follow a rigorous protocol of training, monitoring and double-checking. Until more information about the treatment's risks becomes available, physicians say they will be cautious about prescribing it. On Monday, Hoffman received an e-mail message saying that Tysabri is on its way to the only approved distributor and 12 specialty pharmacies allowed to sell it. That means Hoffman could get a Tysabri infusion at her physician's office within five to 10 weeks. The possible risks and side effects spelled out in the Biogen e-mail gave her some pause, Hoffman said. "But I still want to get back on it," she said. Tysabri works by stopping infection-fighting T-cells from entering the brain, where they can cause the inflammation and scarring that progressively reduce MS patients' mobility and muscle control. No other available MS treatment works as well, analysts and physicians say. But its potency comes at a price: T-cells are an important part of the immune system. By interfering with them, Tysabri makes MS patients vulnerable to herpes infections and PML, a viral infection that typically leads to death or severe disability. Biogen also acknowledged that patients receiving Tysabri have come down with other atypical infections. The possible side effects pose a dilemma for physicians. "Everybody is excited, but also a little concerned," said Dr. Mitch Freedman of Raleigh Neurology. The practice treats about 2,000 MS patients. Hoffman is among about a dozen patients Freedman plans to put back on Tysabri. That's a fraction of the 100 patients who were lined up for a prescription 18 months ago, Freedman said. Dr. Ugo Goetzel of Millennium Neurology in Durham has whittled the number of patients who will receive Tysabri to a handful. Both physicians agreed it is wise to limit the drug to patients who cannot tolerate other MS treatments or whose symptoms have gotten worse despite treatment with other drugs. Hoffman went back on Avonex, another Biogen MS drug, when Tysabri was pulled. But Avonex made her very tired and gave her flu-like aches and pains the day after each weekly injection, she said. Diagnosed with MS 18 years ago, Hoffman works out in the gym daily to keep the stiffness in her legs from getting worse. Despite the exercise, the disease advanced while she took Avonex, she said. Hoffman said she felt different after the only Tysabri infusion she received, in January 2005. "I felt absolutely wonderful," she said. "I kept waiting for something to happen, but no adverse reactions whatsoever. Then they pulled it." The conditions that Biogen, Elan and regulators agreed on to bring Tysabri back are extensive. They include close patient monitoring for signs of PML. Physician offices must receive training before they can prescribe and administer Tysabri infusions. Prescription refills must be authorized by Biogen every six months. Also, Tysabri should not be given to patients with weakened immune systems. Despite the safeguards, Freedman and Goetzel said they remain uneasy about Tysabri. They said they will feel better once more data on Tysabri is available from the monitoring program. "We'll get more answers over time," agreed Eric Schmidt, an analyst with SG Cowen who tracks Biogen. Until then, he and other analysts project annual Tysabri sales at $100 million to $400 million. A year's supply will cost $28,400, up about 20 percent from the introductory price 18 months ago. Schmidt projected that sales of Tysabri could reach $1 billion a year, but not before 2010. "No one would predict this to be a drug as big as it was once conceived to be," he said. Biogen still cautious The same caution prevails at Biogen, which has brought back some of the 650 jobs it cut as part of a companywide restructuring in September. The company employs about 670 on its RTP campus, up about 110 from about 10 months ago. But the majority of the jobs were added because Biogen moved customer support positions from other locations. Biogen's RTP campus is home to manufacturing facilities that produce Tysabri, Avonex and Amevive, a psoriasis treatment. As part of customer support, the company also runs a call center in RTP to respond to patients' questions. As she waits for Tysabri, Hoffman wears her red bracelet every waking hour. She stopped taking Avonex in June after the Food and Drug Administration allowed Tysabri to be sold again. But she hasn't heard from Freedman's office about when she can start the medical checks and the paperwork that are required to get a prescription. "I'm just waiting to get the phone call," Hoffman said. |