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Update on Lemtrada Approval

In December of last year the FDA denied approval of Sanofi’s new MS drug Lemtrada. John Marler, a drug reviewer for the FDA, sited unclear benefits of Lemtrada and potential side effects as the reason for the denial. According to the FDA it was not clear from the clinical trials if the risks associated with Lemtrada outweighed the potential side effects. They also added that the benefits of Lemtrada would have to be “substantial” in order to justify exposing patients to the potential side effects. Lemtrada’s developers argue that side effects that occurred in clinical trials were all non life-threatening, treatable conditions. During clinical trials two deaths occurred, one from sepsis and one from accidental causes.

Two Phase III clinical trials were done comparing Lemtrada to interferon injections. The FDA was concerned about the design of these studies, citing that they were not blind studies which can potentially bias the results. This means that the patients and the doctors knew if Lemtrada or interferon was being given. Blind studies mean that neither the patient or the doctor knows which treatment is being given. This is the preferred design of a clinical trial because the attitudes of the doctor and the patient, whether it be skepticism or enthusiasm, has been shown to skew data. However, in the case of these studies a blind comparison would be almost impossible because one drug is an injection given three times a week and one is an IV infusion given one year apart. The only way I see a blind study happening is if they compare Lemtrada infusions to placebo infusions. Unfortunately doing a placebo study would mean that half of the MS patients in the trial would be going without any treatment for at least two years.

Since the denial I have been waiting to hear what Sanofi, who acquired Genzyme, is going to do next. The FDA asked Sanofi to do another Phase III study with a different design. However last month Sanofi’s CEO Chris Viehbacher publicly stated that paying for and carrying out a new study is out of the question. Instead Lemtrada’s development team is working on a report that they will be submitting to the FDA later this quarter. In it they are extending the data from the existing trials to show results over a longer period of time, and to hopefully convince the FDA of Lemtrada’s potential benefits. They are hoping this is enough to get FDA approval.

Lemtrada is given via IV infusion, and its dosing is unique compared to current treatments. The first treatment is given IV once a day for five days, then another round lasting three days is given one year later. Researchers think that most patients will only require two doses in order to gain a long term benefit. Like other infusion medications already on the market it would be used on patients for whom other treatments have failed. It would also have to be given under the care of a specialist and, similar to other MS drugs, side effects would have to be monitored closely.

Campath, which is the same drug as Lemtrada, is used to treat leukemia. It has been taken off of the market in the US by Genzyme Sanofi while they have been attempting to gain FDA approval for Lemtrada. This has been to prevent doctors from prescribing Campath off label for MS patients. So currently patients who have progressive disease and are desperate for another option to try, have no way of gaining access to this potentially effective treatment. Taking Campath off of the market and rebranding it as Lemtrada, an MS drug, would mean that Genzyme Sanofi would go from making almost no profit to making roughly $60,000 per treatment.

So clearly Genzyme Sanofi stands to make a significant financial gain from Lemtrada, and patients gain the opportunity to try another treatment. After reading the clinical studies it is clear that Lemtrada is not without risks, but I don’t see how this is so different from every other disease modifying drug on the market. It will be interesting to see what happens next.

This article represents the opinions, thoughts, and experiences of the author; none of this content has been paid for by any advertiser. The MultipleSclerosis.net team does not recommend or endorse any products or treatments discussed herein. Learn more about how we maintain editorial integrity here.

  1. http://www.fiercebiotech.com/story/sanofis-ms-team-rebuilds-its-case-fda-rejected-ms-drug-lemtrada/2014-04-30
  2. http://www.medscape.com/viewarticle/818420
  3. http://www.medpagetoday.com/clinical-context/MultipleSclerosis/35702

Comments

  • monarch
    5 years ago

    FDA asked for comments with a direct link to respond, they never even confirmed receipt of my response.Below is my response to the initial FDA denial.

    The FDA’s rejection rationale for MS therapy Lemtrada deserves a through rebuttal. That rebuttal requires a deeper look at each individual responsible for the FDA decision. Then ask why Europe, Canada, Australia approved. Europe has the world’s highest prevalence per 100,000. Furthermore, medical experts using real world patient data supported Lemtrada’s approval. Lemtrada’s risk and cost compared to tangible real benefits was a factor driving approving countries.

    I was motivated to respond to the FDA even if it won’t be complete. Furthermore I have Multiple Sclerosis so for me even these small efforts to organize my thoughts can be a struggle. It’s easy to rationalize putting off my response but a challenge is needed. So having said that let me make a few comments. I hope time is available (FDA deadlines) and personal energy to submit a more complete rebuttal.
    I’m a MS patient on Tysabri. Unfortunately I remember the FDA removed Tysabri twice while patients were benefiting.
    The rationale was illogical and motivation questionable. In my opinion it was lack of initiative and patient concern that was absent to recognize what was best as an aggregate. Patients made their feelings known to the FDA. Those patients would assume potential risks. A basic freedom we often assume is real now removed by a few individuals with no transparency into their motivation.
    Tysabri was and is safe. Why because by combining close observations via frequent office visits, blood work to test for JVC virus and semiannual MRI’s has made risk of PML statistically nonexistent. This rigorous observation will be with Lemtrada. FDA used Europe’s cases with PML to immediately rationalize removing Tysabri. As you must know MS tysabri users were mixing treatments with Avonex coupled with other unrelated Tysabri factors. The initial lack of rigor with MRI, blood work can be assumed to be potential contributing factors. But at the end of the day it’s the patient’s decision with his or her medical advisors of choice. Not a hand full of federal employees. The key word is freedom of choice.

    Every advancement or life enhancing product is the direct result from freedom of choice. Its individual risk taking using their chosen “consultants” then followed by trial and error period that leads to an improved product for society. Anyone can observe this phenomenon with the internet or software that has dramatically improved our lives. At first Software changes are never perfect or even worth the effort and money. It’s without fail there will be frustration, unexpected substantial costs incurred, customers lost, and faulty conclusions drawn that negatively impact lives of other people. But this is the unavoidable process of continuous improvement as feedback from those informed risk takers are part of creating a better solution/result that benefits everyone.
    Tesla, Iphone, Kahn Academy, Microsoft, or Pharmaceuticals latter found to provide other uses. For example, Propecia originally used to treat Prostate enlargement now used for male pattern baldness. Viagra prescribed for heart disease now used for erectile dysfunction. You get the idea.

    I don’t know who or how many FDA employees have hands on MS neurological expertise were directly responsible for the rejection of Lemtrada. Those individuals are in the minority to the rest of the world that see benefits and rationale for choice.

    In conclusion we must consider human bias, massive industry pressure, political influence, lack of expertise/experience unfortunately concentrated in the hands of a few federal employees. Existing FDA MS approved treatments are concentrated in about 3 or so companies.

    Its rationale to ask do FDA decision makers have connections, ever worked with or received funding from these few MS drug companies. The FDA process is long overdue for independent auditing and creating more public transparency. Pressure not to approve could come from Biogen that in Feb 2013 paid 3.25 Billion to receive the remaining drug rights for Tysabri from Elan. Biogen also sells the FDA approved MS therapy Avonex. The life time cost using Lemtrada versus Tysabri or Avonex is not only less expensive but has potential to be a life altering drug for MS patients and society.

    Please reconsider your decision.

    Respectfully but emotionally involved
    John DiStanislao

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