A new treatment option: Tysabri returns, with controls.

On June 5, the U.S. Food and Drug Administration (FDA) released Tysabri back to market for people with relapsing MS, despite its risk of causing PML, a potentially fatal disease. To minimize this risk, the drug will be available only at authorized infusion centers using a rigorous monitoring program.

Tysabri (natalizumab), which is given as a monthly IV infusion, was withdrawn in 2005 by Biogen Idec and Elan Pharmaceuticals, Inc., after three people among the 3,000 who had been taking the drug in clinical trials developed PML (progressive multifocal leukoencephalopathy). Two of them died.

But last March at a special hearing before an FDA advisory panel, dozens of people affected by MS testified about what Tysabri meant to them. The panel evaluated data suggesting that Tysabri may have twice the efficacy rate of other MS treatments. After reviewing additional safety studies and a proposed risk management plan, the panel recommended the drug's return. That recommendation is now formally accepted. The approval includes a mandatory registration program for patients and their prescribing physicians. The drug also carries a "black box" warning describing the risks.

A new option added to our list

"It's important that people with relapsing MS now have a new treatment option," said John R. Richert, MD, vice president of Research and Clinical Programs for the Society.

"Because of the risk of PML," Dr. Richert continued, "the FDA is recommending that Tysabri be given only to people who have had an inadequate response to, or are unable to tolerate, the other MS therapies--Copaxone, Betaseron, Avonex, Rebif, or Novantrone. But they leave it to individual physicians and patients to define 'inadequate response' and what it means to be 'unable to tolerate' other therapies, giving physicians and people with MS leeway to exercise their best judgment.

"We believe that the mandatory patient registry and the post-marketing observational study will help clarify the potential risks and benefits of this new therapy for people with MS," Dr. Richert said.

Biogen and Elan's post-marketing study will follow at least 5,000 people prescribed Tysabri for five years to evaluate the long-term safety. Dr. Richert urged people considering any MS treatment to discuss the options with their health-care professional.

Tysabri: a few answers

Who should take Tysabri?

Tysabri is approved for people with relapsing forms of MS who do not have another condition that weakens the immune system, such as cancer or lupus.

It is FDA-approved as a monotherapy. It cannot be used with Avonex, Betaseron, Copaxone, Rebif, Novantrone, monthly IV steroids or other immune modulating drugs.

How is Tysabri given?

Tysabri is given once a month by intravenous infusion, only at an authorized infusion center.

What is the primary safety concern regarding Tysabri?

People taking Tysabri risk developing PML.

How will safety risks be managed?

At all infusion centers, physicians and patients will be registered in a mandatory program called TOUCH. Medical personnel will receive special training in recognizing early signs of PML. Information about all the risks and side effects will be given to each patient. Before every infusion, medical staff and patients will complete a checklist for signs of PML and other side effects.

Is Tysabri available everywhere?

Biogen Idec began supplying Tysabri to infusion centers in July, and are continuing to train healthcare professionals and register new centers around the country. For information about centers, contact Biogen Idec.

How expensive is Tysabri?

The annual wholesale cost of Tysabri is $28,400. There will be additional charges for infusion services.

Will insurance cover the cost?

There may be a lag time before the drug is included in private health plans. Prior authorization will probably be necessary for most health plans.

Tysabri is expected to be covered by Medicare Part B because it is given at a medical facility.

People with Medicaid should check with their state program. There may be a delay before Tysabri is included in the Medicaid preferred drug list.

More information is available three ways:

1. From us at www.nationalms society.org/tysabri or call your chapter at 1-800-FIGHT-MS (1-800-344-4867)

2. From the FDA at www.fda.gov/cder/druglinfopage/natalizurnab/defaul.thtm

3. From Biogen Idec at www.biogenidec.com or call 1-800-456-2255.