Archive for: November, 2004

First Monoclonal Antibody Treatment For Multiple Sclerosis Approved

FDA today licensed a new biologic approach to treat patients with relapsing forms of multiple sclerosis (MS) to reduce the frequency of symptom flare-ups or exacerbations of the disease. MS is a chronic, often disabling disease of the brain and spinal cord.

Natalizumab, the new product, is a monoclonal antibody bioengineered from part of a mouse antibody to closely resemble a human antibody. It is being marketed under the trade name Tysabri. The product is given intravenously once a month in a physician’s office.

According to the Multiple Sclerosis Association of America, approximately 350,000 individuals have been diagnosed with MS in the U.S., with an estimated 10,000 new cases diagnosed each year. The most common form of MS at the time of initial diagnosis is a relapsing-remitting form, in which acute symptoms or worsening of neurologic function (referred to as “relapses,” “attacks,” or “exacerbations”) occur intermittently. The symptoms can diminish or disappear for months or years between relapses.

Although the cause of MS is unknown, it is widely considered to be an autoimmune disease in which the person’s immune system attacks the brain and/or spinal cord. Tysabri appears to work by binding to these immune system cells, thus preventing them from traveling to the brain where they can cause damage.

Antibodies are proteins produced by a person’s immune system to fight foreign substances, such as infections. Monoclonal antibodies, such as natalizumab, can be produced in large quantities in cell culture in a laboratory setting. They can be designed to bind to proteins on the body’s normal cells. By recognizing and attaching to these proteins, monoclonal antibodies can interfere with (or alter) normal or abnormal cellular responses. In this way, monoclonal antibodies may be useful in the treatment of certain diseases such as MS.

“This innovative treatment for multiple sclerosis represents a new approach to treating MS — exciting news for patients with this serious disease,” said Dr. Lester M. Crawford, Acting FDA Commissioner. “While we eagerly await long-term results from ongoing clinical trials, we have reason to believe that Tysabri will significantly reduce relapses in MS.”

The approval of Tysabri is based on positive results seen in patients after one year of treatment. This product received accelerated approval because it appears to provide substantial benefit for patients with a serious disease. As part of that approval, the manufacturer has committed to continuing its trials of this product for another year.

Tysabri was evaluated for safety and efficacy in two ongoing randomized, double-blind, placebo-controlled trials in patients with relapsing forms of MS. In the first clinical trial of the product’s safety and efficacy, the drug reduced the frequency of relapses by 66 percent relative to placebo.

In a second trial, patients who had been treated with Avonex (interferon beta-1a), an approved treatment for MS, but who had experienced one or more relapses while on Avonex, were randomized to receive Tysabri or placebo. Avonex was continued throughout the study for both groups. In this trial, natalizumab reduced the frequency of relapses by 54 percent relative to placebo.

The most frequently reported serious adverse reactions were infections, including pneumonia, temporary hypersensitivity reactions (such as rash, fever, low blood pressure, and chest pain), depression, and gallstones. These serious adverse reactions were uncommon. Common adverse reactions were generally mild and included non-serious infections (such as urinary tract, lower respiratory tract, GI system, and vaginal infections), headache, depression, joint pains, and menstrual disorders.

Tysabri is marketed by Biogen Idec, Inc., of Cambridge, Massachusetts, and Elan Pharmaceuticals, Inc., of Dublin, Ireland.

Tysabri PML Lawyer

If you or a family member has been diagnosed with PML, or been treated with Tysabri infusions, contact our law firm to speak to a PML lawyer about your legal options. Every attorney at our law firm is dedicated to fighting for the rights of those that have been harmed by dangerous and recalled drugs and medical devices. For over 25 years, Attorney David Willis has been a trial lawyer representing seriously injured clients in product liability and personal injury lawsuits such as defective drug litigation, class action drug lawsuits and medical device recall lawsuits. If Tysabri has harmed you, or a loved one, contact us right now to discuss your potential case in filing a Tysabri PML Lawsuit with an Attorney.

  1. What is natalizumab?
    Natalizumab is a monoclonal antibody that binds to a protein called alpha-4-integrin. Integrins are found primarily on the surface of white blood cells, and play a role in immune system activity.
  2.  

  3. What are monoclonal antibodies?
    Antibodies are naturally occurring proteins produced by the immune system in response to foreign substances. Once produced by the body, they recognize and bind to specific proteins (antigens) on bacteria, viruses, and toxins, to help the body fight disease.

    Monoclonal antibodies, such as natalizumab, are produced in cell culture systems. They can be designed to bind to receptors on the body’s normal cells. By recognizing and attaching to these receptors, monoclonal antibodies can interfere with (or alter) normal or abnormal cellular responses. In this way, monoclonal antibodies may be useful in the treatment of certain diseases.

  4.  

  5. What is natalizumab used to treat?
    Natalizumab is approved to treat patients with relapsing forms of multiple sclerosis to reduce the frequency of exacerbations. It was approved based on results achieved after approximately one year of treatment in ongoing controlled trials. These trials will continue for another year. At this time, the safety and efficacy of natalizumab beyond one year are not known.

    Safety and efficacy in patients with chronic progressive multiple sclerosis have not been established.

  6.  

  7. What is multiple sclerosis?
    Multiple sclerosis (MS) is a disease in which the immune system attacks the person’s brain and spinal cord. The disease causes a wide range of symptoms including fatigue, difficulty walking, numbness, and vision problems.
  8.  

  9. What is relapsing MS?
    This type of MS is characterized by relapses (also known as exacerbations or attacks) during which new symptoms can appear and old ones resurface or worsen. The person fully or partially recovers from the deficits acquired during the relapse. Relapses are followed by periods of remission, during which no new symptoms occur.
  10.  

  11. How does natalizumab work?
    White blood cells are thought to play a major role in causing the damage to the nervous system in MS. Natalizumab binds to white blood cells and interferes with their movement from the bloodstream into the brain and spinal cord.
  12.  

  13. Is natalizumab a cure for MS?
    No, natalizumab will not cure MS. In a study in which patients were randomly assigned to treatment with either natalizumab or placebo (an inactive substance), natalizumab reduced the average yearly relapse rate from 0.74 relapses per patient in the placebo group, to 0.25 relapses per patient in the natalizumab treated group. This is a relative reduction of 66%.
  14.  

  15. What is accelerated approval?
    Accelerated approval is a program the FDA developed to make new drug products available for serious or life threatening diseases when they appeared to provide a benefit over available therapy (which could also mean there was no existing effective treatment). Under this program, natalizumab is being approved on the basis of early clinical study evidence (such as data from only one year of study) suggesting that the drug is reasonably likely to have a valuable effect on symptoms.

    Several other products are already available for the treatment of MS, including Betaseron, Avonex, Rebif, Copaxone, and Novantrone. One of the clinical studies with natalizumab was performed in patients already being treated with Avonex. The addition of natalizumab resulted in a further reduction in the occurrence of relapses, beyond the benefit that those patients had already received from Avonex.

    The approval is granted on the condition that the manufacturer must continue testing to demonstrate that the drug indeed provides therapeutic benefit to the patient. If it does not, the FDA can withdraw the product from the market more easily than usual.

  16.  

  17. How is natalizumab given?
    Natalizumab is given by intravenous (in to a vein) infusion every four weeks.
  18.  

  19. What are the possible side effects of natalizumab?
    The most frequently reported serious side effects with natalizumab are infections, severe or life threatening allergic reactions, depression (including thoughts of suicide) and gallbladder problems. In the trials, these serious side effects occurred at a rate of 0.8% for serious depression and gallbladder problems, at a rate of 2.1% for serious infections.

    Common side effects include:

    • Infections
    • Headache
    • Fatigue
    • Depression
    • Joint pain
    • Menstrual disorders

    A complete list of side effects may be found in the product labeling.

  20. Where can I find more information on natalizumab and MS?
    Go to the FDA’s or the National Library of Medicine’s website.
  1. What is Tysabri?
    Tysabri is a monoclonal antibody that binds to a protein called alpha-4-integrin. Integrins are found primarily on the surface of white blood cells, and play a role in immune system activity.
  2.  

  3. What are monoclonal antibodies?
    Antibodies are naturally occurring proteins produced by the immune system in response to foreign substances. Once produced by the body, they recognize and bind to specific proteins (antigens) on bacteria, viruses, and toxins, to help the body fight disease.

    Monoclonal antibodies, such as Tysabri, are produced in cell culture systems. They can be designed to bind to receptors on the body’s normal cells. By recognizing and attaching to these receptors, monoclonal antibodies can interfere with (or alter) normal or abnormal cellular responses. In this way, monoclonal antibodies may be useful in the treatment of certain diseases.

  4.  

  5. What is Tysabri used to treat?
    Tysabri is approved to treat patients with relapsing forms of multiple sclerosis to reduce the frequency of exacerbations. It was approved based on results achieved after approximately one year of treatment in ongoing controlled trials. These trials will continue for another year. At this time, the safety and efficacy of Tysabri beyond one year are not known.

    Safety and efficacy in patients with chronic progressive multiple sclerosis have not been established.

  6.  

  7. What is multiple sclerosis?
    Multiple sclerosis (MS) is a disease in which the immune system attacks the person’s brain and spinal cord. The disease causes a wide range of symptoms including fatigue, difficulty walking, numbness, and vision problems.
  8.  

  9. What is relapsing MS?
    This type of MS is characterized by relapses (also known as exacerbations or attacks) during which new symptoms can appear and old ones resurface or worsen. The person fully or partially recovers from the deficits acquired during the relapse. Relapses are followed by periods of remission, during which no new symptoms occur.
  10.  

  11. How does Tysabri work?
    White blood cells are thought to play a major role in causing the damage to the nervous system in MS. Tysabri binds to white blood cells and interferes with their movement from the bloodstream into the brain and spinal cord.
  12.  

  13. Is Tysabri a cure for MS?
    No, Tysabri will not cure MS. In a study in which patients were randomly assigned to treatment with either Tysabri or placebo (an inactive substance), Tysabri reduced the average yearly relapse rate from 0.74 relapses per patient in the placebo group, to 0.25 relapses per patient in the Tysabri treated group. This is a relative reduction of 66%.
  14.  

  15. What is accelerated approval?
    Accelerated approval is a program the FDA developed to make new drug products available for serious or life threatening diseases when they appeared to provide a benefit over available therapy (which could also mean there was no existing effective treatment). Under this program, Tysabri is being approved on the basis of early clinical study evidence (such as data from only one year of study) suggesting that the drug is reasonably likely to have a valuable effect on symptoms.

    Several other products are already available for the treatment of MS, including Betaseron, Avonex, Rebif, Copaxone, and Novantrone. One of the clinical studies with Tysabri was performed in patients already being treated with Avonex. The addition of Tysabri resulted in a further reduction in the occurrence of relapses, beyond the benefit that those patients had already received from Avonex.

    The approval is granted on the condition that the manufacturer must continue testing to demonstrate that the drug indeed provides therapeutic benefit to the patient. If it does not, the FDA can withdraw the product from the market more easily than usual.

  16.  

  17. How is Tysabri given?
    Tysabri is given by intravenous (in to a vein) infusion every four weeks.
  18.  

  19. What are the possible side effects of Tysabri?
    The most frequently reported serious side effects with Tysabri are infections, severe or life threatening allergic reactions, depression (including thoughts of suicide) and gallbladder problems. In the trials, these serious side effects occurred at a rate of 0.8% for serious depression and gallbladder problems, at a rate of 2.1% for serious infections.

    Common side effects include:

    • Infections
    • Headache
    • Fatigue
    • Depression
    • Joint pain
    • Menstrual disorders

    A complete list of side effects may be found in the product labeling.

  20. Where can I find more information on Tysabri and MS?
    Go to FDA’s website or the National Library of Medicine’s website.

Tysabri PML Lawyer

If you or a family member has been diagnosed with PML, or been treated with Tysabri infusions, contact our law firm to speak to a PML lawyer about your legal options. Every attorney at our law firm is dedicated to fighting for the rights of those that have been harmed by dangerous and recalled drugs and medical devices. For over 25 years, Attorney David Willis has been a trial lawyer representing seriously injured clients in product liability and personal injury lawsuits such as defective drug litigation, class action drug lawsuits and medical device recall lawsuits. If Tysabri has harmed you, or a loved one, contact us right now to discuss your potential case in filing a Tysabri PML Lawsuit with an Attorney.