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Pomalyst (Pomalidomide) Approved By FDA For Relapsed And Refractory Multiple Myeloma

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Published: Feb 8, 2013 3:50 pm; Updated: Feb 8, 2013 9:20 pm

The United States Food and Drug Administration (FDA) has approved pomalidomide, which will be marketed under the brand name Pomalyst, for the treatment of relapsed and refractory multiple myeloma.

Specifically, Pomalyst has been approved for use in patients with multiple myeloma who have received at least two prior therapies including Rev­limid (lenalido­mide) and Velcade (bortezomib) and have demon­strat­ed disease progression on or within 60 days of completion of the last therapy.

Celgene (NASDAQ:CELG), the company that will market Pomalyst, has told The Beacon that the drug will be available in U.S. pharmacies in about two weeks.

Pomalyst is the second drug, following Kyprolis (carfilzomib), that the FDA has approved for the treatment of multiple myeloma over the past year.  Prior to Kyprolis, the last FDA approval of a novel myeloma therapy was in June 2006, when the agency approved Revlimid as a treatment for myeloma.

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Pomalyst also is being reviewed by the European Medicines Agency for approval in Europe. Celgene submitted the European marketing authorization application for the drug in June of last year.  In recent discussions with financial analysts, the company has said that it expects a decision on this application by the second half of this year.

Pomalyst is chemically similar to Revlimid and thalidomide.  All three drugs are administered orally as a capsule or tablet, and the three drugs together make up the class of myeloma therapies known as immunomodulatory agents.

Celgene, which developed Pomalyst and will sell it in the U.S., also markets Revlimid and thalidomide in the U.S. and internationally.

The FDA’s decision regarding Pomalyst’s new drug application is likely to be its last approval of a new myeloma therapy for at least a year.  There are a number of potential new myeloma therapies in the later stages of development, including elotuzumabpanobinostat, and perifosine.  However, many analysts believe it may not be until 2015, and possibly even later, until one of these drugs becomes the next novel anti-myeloma therapy to gain FDA approval.

Celgene has not yet said publicly what the price of Pomalyst will be.  However, it has confirmed with The Beacon published reports that Pomalyst will cost about $10,500 per 28-day cycle, based on a 21-out-of-28-day dosing regimen.

At that price, Pomalyst will be the most expensive treatment for multiple myeloma.

In comparison, Revlimid costs $8,400 per 28-day period at the FDA-approved 21-out-of-28-day dosing; Velcade costs between $4,100 and $8,200 per 28-day period, depending on the frequency of dosing; and Kyprolis costs $10,000 per 28-day cycle at the recommended dose for a patient of average size.

Pomalyst’s FDA approval is based on data from the Phase 2 “MM-002” clinical trial of Pomalyst plus dexamethasone in relapsed and refractory myeloma patients.

Updated results from this study were presented at the American Society of Hematology (ASH) meeting in December (see related Beacon news).  They showed that the combination of Pomalyst and dexamethasone had an overall response rate of 34 percent in myeloma patients with a median of five previous lines of therapy.  Median progression-free survival in these patients was 4.6 months and median overall survival was 16.5 months.

Also presented at the ASH meeting were initial results of the Phase 3 “MM-003” clinical trial comparing Pomalyst plus low-dose dexamethasone to only high-dose dexamethasone, also in heavily pretreated myeloma patients.  These results showed that the combination of Pomalyst and dexamethasone had a statistically significant overall survival advantage compared to treatment with with dexamethasone alone (see related Beacon news).

In Pomalyst’s clinical trials, the most frequent serious side effects were low red blood cell, white blood cell, and platelet counts, as well as infections such as pneumonia.

A number of additional clinical trials involving Pomalyst as a treatment for multiple myeloma are currently ongoing.  All involve relapsed/refractory patients who have been previously treated with Revlimid, and all involve Pomalyst used in combination with dexamethasone and at least one other drug, such as Velcade, Kyprolis, or Doxil (doxorubicin liposomal).

Additional details about today’s FDA approval of Pomalyst are available in press releases from Celgene and the FDA and the Pomalyst prescribing information.

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20 Comments »

  • Jan Stafl MD said:

    All right! We in the MM community have been waiting for this day. I am so glad that this important option is approved today. Now recurrent MM patients have another great option to consider, along with Kyprolis approved last year. Now on to further immunotherapy option research, especially the monoclonal antibodies and vaccines. Thank you for the news! Jan

  • Michelle said:

    Too late for my husband Tom who lost his 13 month battle with MM 4 months ago. He had extra medullary progression and the SCT kept it at bay for only 53 days.

  • Ron Harvot said:

    Great that we have another arrow in quiver to fight the disease but the cost is outrageous. $10,500 dollars for 21 pills or $500 per pill!!! A one year treatment $126,000!!!

    I wonder what the insurance industry’s response will be.

    The problem with MM is that it is a relatively small number of patients that will be taking this drug. The costs of development, testing, FDA approval etc. have to passed on to this small group. That is where we need government subsidising of the expense and a cap on the charges. What good is new drug if the patients cannot afford it.

    Ron

  • Jan Stafl MD said:

    Michelle, I’m so sorry for your loss. And Ron, you make good points. I realize that I and my insurance company subsidize many who cannot afford these meds. Due to budget deficits, the US government cannot afford that. BTW, Revlimid and Kyprolis are just as expensive. It is easy to say that we cannot put a price on human life, but realistically it is not unlimited. As a physician, I support universal health care for all, which is the ONLY way that the risk can be spread across the entire population. Every other industrialized country does, if for no other reason than the fact that it is cheaper for society as a whole. Obamacare has many problems, but hopefully that will a stepping stone to universal coverage with less profit motive that I believe is running US health care. We need political will to make this happen, that can resist the many special interests in the way. Also, an emphasis on preventive and holistic medicine can be a fiscal benefit and a life saver! Just my thoughts on this momentous day…. Jan

  • Kayitesi Karasira Cecile said:

    I am a patient with multiple myeloma and I am so happy to learn about this new drug in the treatment of the disease.I was treated with thalidomide at first but developed complications. This was stopped after taking it for only five months.I was then given two and half cycles of bortezimib from which I developed grade 3/4 peripheral neuropathy.The disease affected my vertebrae discs such that I was operated upon and had discectomy with fixtures in situ.This is my third year in remission but have very bad bone pains again. my doctor in India has put me on 10mgs since Dec last year which I m not able to take well as it makes me weak with diarrhea and headaches. I am from Rwanda in Africa . I cannot manage to go back to India as my finances are exhausted. I was in preparation for bone marrow transplant after 9months and I cannot afford the expenses. How can u help me get this new drug. Please consider me for this new drug, I appeal to u. Thank u.

  • nancy shamanna said:

    Hi Kayitesi, i am sorry to hear that you have been suffering from multiple myeloma and that you haven’t found enough treatment yet to get you much better. it seems complicated that your oncologist is in India and yet you live in Rwanda. Is it possible to switch to a local oncologist in Rwanda who might better be able to treat you there? When you say that you were on 10 mgs of a drug, what drug was that? There is now a capability for bortezimib to be administered by sub-cutaneous routes, which is meant to reduce neuropathy.

    Pomalyst is as yet only approved in the US. i think that have international ‘compassionate care ‘ programs at the parent company, and maybe you could write to them directly, with the help of your doctors.

    I hope that this helps you! Best wishes to you.

  • Ron Harvot said:

    Jan,

    I am in total agreement with you concerning universal coverage. Nobody should face financial ruin because of health care costs. As things currently stand, I will have to work as long as I am physically able just to keep my health care coverage. I just hope I can stay in remission and do that. Even with medicare the costs are high.

    Ron

  • nancy shamanna said:

    Of course we like universal health care coverage in Canada. Although it is not perfect, and I suppose that no system is really, at least our health costs are covered. Cancer patients very often still suffer financially due to inability to work, etc., but we can at least rely on our cancer centres to treat us equally. The social ‘safety net’ is something we are used to having here.

  • tony said:

    at 10k a month. what a bargain, dont you think, with a response rate of less than 10% a single agent. give me a break

  • Jan Stafl MD said:

    Thank you Ron. Nancy, since my wife Líba is Canadian, our three children are dual US and Canadian citizens. Our 25 year old identical twin daughters have chosen to live in Canada (Vancouver and Canmore AB), due to the great outdoors, but also due to the security of health care, which is one factor why the Canadian economy is healthier than in the US for over 5 years now.

    Tony, I understand how underwhelmed you are by the statistics with Pomalyst. However, this medication will be used in active treatment of recurrent MM in various combinations using Velcade, Kyprolis, dex, or Biaxin, for which response rates in heavily pretreated patients is well over 50%, even in high risk patients. If you face this situation, it is welcome news indeed before resorting to investigational meds or allo transplants. The addition of elotuzumab or other meds will likely increase response rates significantly. I believe many hem-onc. physicians will choose combination therapy using Pomalyst and/or Kyprolis as initial novel agent induction chemotherapy for high risk patients: that’s the “hit it hard and fast” philosophy. Creative combinations using these novel agent therapies will no doubt be studied in level 3 trials.

    But you are right, all of these are likely to present only 1-2 year OS benefit, so other approaches (especially immunologic) are sorely needed. The expense is a major hurdle, but the research is expensive, and our system has to encourage research financially in order for the pharmaceutical companies to continue to invest in MM and other research. An international effort is needed, and in fact is occurring on all continents (Antarctica excluded :).

    So that is a rather long winded explanation of the dilemma of MM research. Many clinical decisions in MM treatment are still empirical, with the experience and regional bias of specialists, along with patient choice (and insurance), being the main variable in what treatment is chosen. Caveat emptor! Get educated in all your options everyone! Jan

  • nancy shamanna said:

    Hi Jan, Hope that your daughters are enjoying living in Western Canada. It has been quite a cold winter in Alberta, and the mountains do share that weather. We had a nice weekend in Canmore over the holidays. I was trying out new snowshoes since I don’t feel confident skiing any more! (i can’t take the falls into the snowbanks like i used to!) You should send me a message if they are going to be in Calgary.

    I don’t know that our economy was doing better due to our health care system recently, but I think it is true that it cuts down on paperwork and administration costs to have one system. Each province does run their own health care system though, since that is a provincial responsibility, and that is where the majority of the funding comes from!

  • Ron Harvot said:

    “The expense is a major hurdle, but the research is expensive, and our system has to encourage research financially in order for the pharmaceutical companies to continue to invest in MM and other research.”

    One way that the government can “subsidize” the research is through the Research Credit. By making the credit “refundable” needed cash can flow back to the companies. As the credit stands now it is very complicated to obtain and maintain. Furthermore it is not refundable. The company’s can only offset tax from other profit with the credit. If it was made refundable then when research costs exceed net taxable income they could still get the cash to help offset the actual reserch costs.

    As you can tell I am a tax guy.

    Ron

  • Jan Stafl MD said:

    Hi Nancy, you can find my daughter Lenka at Howling Dog sled dog tours in Canmore, where she guides till 2/22. That would be a great adventure for you and your family! How is the coverage of your meds and overall health care in Alberta compared to BC?

    Ron, you detail a great tax incentive idea! Maybe LLS can lobby for that. Thanks, Jan

  • nancy shamanna said:

    Hi Jan, Thanks for that info! Haven’t been on a sled dog tour, actually, but am having a lot of fun with the ‘grand puppy’.

    i think that the BC/Alta health care is similar, but there is some variation from one province to the next. Vancouver also has a myeloma support group, of that I am aware. Funding for cancer drugs can vary from province to province. First a new drug, such as Pomalyst, must be approved by Health Canada. (And of course, the drug manufacturer must apply for approval here.) Then, it is up to the individual provinces to fund the drug..that can take a long time. I guess we have to access clinical trials, where possible, for access to the newer drugs for the time being.

  • Multibilly said:

    Well, in spite of some of some of the negative comments going on here, I am personally delighted and relieved to see that another option is available to MM patient and myself down the road, should I ever need it. I’m sure many of you have read and follow Don Wright’s blog (“Myeloma Hope”), which is quite inspirational… he sure seems to have done well by the drug.

  • shailja said:

    Hi

    i want to know how and when can it be available in india. My father is in 6th year of disease after 3 remisssion , we critically need a new line of treatment, please do suggest a sure shot way to get this drug.

  • Terry L said:

    I agree somewhat with Multibilly. For all the US health care system’s flaws, we are lucky in the US to have access to these novel therapies which, for the most part, have been developed domestically. Most major cancer centers actually have myeloma departments. Such is not the case in many other countries, including Europe. I received carfilzomib in a clinical trial and thank God I got it. It worked well for me. I also read the Myeloma UK site which has a nice forum and patients there subject to their NHS can only dream of getting access to what we get in the US.

  • Jan Stafl MD said:

    Sure, American medical research and the availability of newly approved medications is the best in the world, IF you are well insured or really well off. It is the most expensive in the world, and the health outcome epidemiology in the US is inferior to many other industrialized countries in numerous ways. It is really like the Tale of Two Cities. So while Pomalyst approval is an important step, many in the USA who need it will not be able to afford it, while patients in other countries need to rely on earlier, less expensive drugs, unless they take part in clinical trials.

    Don Wright’s experience with pomalidomide maintenance while running marathons in all 50 states is inspirational!

  • nancy shamanna said:

    Hi Jan, i think that since the latest new drugs for myeloma were developed in the US (Kyprolis and Pomalyst), and were approved there, it will take some time for them to be approved in other countries. However, I am an optimist by nature and I think we will get them here too! They are very expensive, but so were the drugs I was treated with…Velcade and Revlimid. I am so grateful to our health care system for providing those drugs, plus a stem cell transplant! The treatments basically gave me back my life!

    I follow the business news for companies I find to be interesting…and guess what, Revlimid was approved for use in China yesterday! I wonder what the set up there is for accessibility for myeloma drugs?

  • Terri Dickerson said:

    Hi MM Friends
    My husband has MM that has relapsed three times. Currently Cytoxan/Dex/Velcade. Once he reaches remission again his doctor wants to try Pomalyst. He has excellent insurance coverage. Beacon article says cost to be about $10,500 per 28 day cycle. However, I just checked his AARP insurance. They are showing a cost per year of: We pay – $213,647.82 Insurance Pays – $130,886.64. Mfr. pays – $4,671.13. This is OUTRAGEOUS! Who can possibly afford $213K out of pocket?