Pomalyst – Which Multiple Myeloma Patients Will Physicians Treat With It?
Published: Feb 22, 2013 12:37 pm; Updated: Feb 22, 2013 4:10 pm
Now that Pomalyst has been approved in the United States as a treatment for multiple myeloma, the drug is available for physicians in the U.S. to prescribe for their patients.
The broad availability of Pomalyst, however, raises the important question: For what types of myeloma patients are physicians likely to prescribe Pomalyst?
Early this month, the U.S. Food and Drug Administration (FDA) approved Pomalyst (pomalidomide) for the treatment of certain multiple myeloma patients. These patients must have received at least two prior therapies, including Revlimid (lenalidomide) and Velcade (bortezomib), and the patients must have demonstrated disease progression on or within 60 days of completing their last therapy.
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Although Pomalyst has been approved by the FDA for myeloma patients who meet these specific criteria, once a drug is approved in the U.S., physicians have substantial freedom to prescribe the drug as they deem appropriate.
At the same time, Medicare, Medicaid, and private insurance companies may decide to control health care costs by restricting reimbursement of Pomalyst treatment to patients meeting the FDA criteria described above. This may particularly be the case with Pomalyst, given that it is currently the most expensive myeloma treatment available.
In addition, the FDA's approval of Pomalyst did not happen in a vacuum. Kyprolis (carfilzomib) was approved by the FDA last summer for myeloma patients previously treated with Velcade and an immunomodulatory agent, such as thalidomide (Thalomid), Revlimid, and now Pomalyst. As is the case with Pomalyst, the FDA approval for Kyprolis is for use in patients who demonstrated disease progression on or within 60 days of completing their last therapy.
So, for which patients are myeloma specialists likely to prescribe Pomalyst? Will they feel they need to choose between Pomalyst and Kyprolis and, if so, how will they choose between the two drugs?
To gain a better sense of what the answers to these questions are likely to be, The Beacon conducted an informal survey with a wide range of myeloma specialists around the country. We asked each of them the following question: “How do you see yourself using Pomalyst in the near future – for example, for what kinds of patients? Feel free to comment on which types of patients may be better candidates for treatment with Pomalyst as compared to Kyprolis.”
A similar survey was conducted after the FDA's approval of Kyprolis to find out what types of patients myeloma specialists expected to treat with Kyprolis at that time (see related Beacon news).
The rest of this article summarizes what the myeloma specialists told The Beacon about their likely prescribing habits now that both Pomalyst and Kyprolis are available.
Please note that some of the feedback The Beacon received has been edited for brevity and clarity. For consistency, the comments also have been edited to use the Beacon's conventions for referring to drug names.
Ruben Niesvizky, Weill Cornell Medical College:
Pomalyst has brought to us yet another great option, particularly in patients who are no longer responding to or have progressed on Revlimid.
As we reported at the 2012 American Society of Hematology (ASH) meeting, Pomalyst in combination with clarithromycin (Biaxin) can yield response rates near 60 percent even in patients with double refractory disease [that is, disease resistant to treatment with Revlimid as well as Velcade]. We are currently using this combination particularly in relapsed and refractory high-risk patients.
With the approval of Pomalyst and Kyprolis, patients with relapsed and refractory disease have more options now. We are eagerly waiting to see the results of ongoing clinical studies looking at potential sequential use of these drugs or in combination.
Philip McCarthy, Roswell Park Cancer Institute:
I will follow the FDA guidelines for Pomalyst (for patients receiving at least two prior therapies including Revlimid and Velcade and who have demonstrated disease progression on or within 60 days of completion of the last therapy).
For Kyprolis, the FDA indication is for multiple myeloma patients receiving at least two prior therapies, including Velcade and an immunomodulatory agent, and have demonstrated disease progression on or within 60 days of the completion of the last therapy. So, there is a subtle difference in that for Kyprolis, the failure is to Velcade and an immunomodulatory drug (Revlimid or thalidomide [or now Pomalyst]) whereas for Pomalyst it is Velcade and Revlimid.
The response rate to Kyprolis alone is about 20 percent, and it is higher when combined with Revlimid and dexamethasone (Decadron). Pomalyst appears to be more effective when combined with dexamethasone, and the expectation is that it will have more activity when combined with a proteasome inhibitor [such as Velcade or Kyprolis].
Thus, it remains to be seen as to the best approach to sequencing these therapies in the treatment of relapsed disease.
As we have a large number of patients who have seen Kyprolis, we will be recommending Pomalyst with dexamethasone for disease progression. For patients who are Kyprolis and Pomalyst naïve, either therapy would be reasonable for salvage realizing that the other drug could be used later. Combination therapy trials will likely show even more benefit based on the recent ASH abstracts.
Vincent Rajkumar, Mayo Clinic:
Pomalyst is an important addition for the treatment of multiple myeloma, and it is very well tolerated. Its main role will be in patients who are refractory to Revlimid (and Velcade). Pomalyst and Kyprolis are both approved for similar indications, but I do not consider them competitors. Patients need both options, and the use of one does not, and should not, preclude the other. As time goes on, I expect both drugs to be tested and used earlier in the disease course.
Ravi Vij, Washington University in St. Louis:
Pomalyst will be used as per label for patients who have received prior treatment with Velcade and Revlimid. Ultimately, nearly all patients will be treated with both Kyprolis and Pomalyst in one sequence or the other and also with combination therapy such as the Pomalyst-Kyprolis-dexamethasone regimen.
Amrita Krishnan, City of Hope:
We are obviously very excited about Pomalyst.
I would say patients will eventually be exposed to both [Pomalyst and Kyprolis] in the course of their illness. Much of it depends on whether you hold the theory that you need to change class of drugs at progression.
The oral route of Pomalyst confers many advantages. We are going to be opening a trial combining it with MLN9708, perhaps getting the best of both worlds in the relapsed and refractory population.
Guido Tricot, University of Iowa Hospitals and Clinics:
The use of Pomalyst and Kyprolis will be limited, since there is limited experience with these drugs when compared to thalidomide/Revlimid and Velcade.
Pomalyst and Kyprolis are not a new class of agents. Kyprolis works like Velcade, and Pomalyst works like thalidomide/Revlimid.
Pomalyst will be tried in patients who cannot be on thalidomide and/or Revlimid because of toxicities of these drugs. Kyprolis will be mainly used for patients who have pronounced neuropathy due to Velcade.
Ken Shain, Moffitt Cancer Center:
I think that the FDA approval of Pomalyst is very exciting for us as physicians, but most importantly for our patients. Our patients now have access to a very exciting and efficacious additional line of therapy for disease control.
For now and off study, Pomalyst needs to be utilized as labeled by the FDA: for patients who have received at least two lines of therapy including Revlimid and Velcade and demonstrated disease progression on or within 60 days of the last day of therapy.
Where in the sequence of therapy Pomalyst should be used has yet to be firmly established. Further, there is a lot of excitement regarding the efficacy of different combination therapies with Pomalyst.
Jason Valent, Cleveland Clinic:
In the short term, I will use Pomalyst in patients who have relapsed disease after treatment with either Revlimid or Velcade with relatively normal kidney function. In patients with abnormal kidney function, I feel that Kyprolis is a better option until further study is done on the correct dosing of Pomalyst in patients with [abnormal kidney function].
Obviously, patient preference for Kyprolis or Pomalyst will be a consideration as they both seem to work well in the relapsed or refractory setting and head-to-head comparisons are not yet available.
Jeffrey Wolf, University of California, San Francisco:
The approval of Pomalyst increases our options for treating patients with myeloma.
For now, it is best used for patients who have progressed on other therapies, but eventually, if it proves better than Revlimid and less toxic, it may move to an earlier position.
The choice of Pomalyst or Kyprolis will depend on prior therapies, toxicities, and responses.
Peter Voorhees, University of North Carolina at Chapel Hill:
Effective now, Pomalyst and dexamethasone should be used as stipulated in the FDA label: for patients who have received at least two prior therapies for their myeloma, including regimens that contain Revlimid and Velcade, and have had disease progression on or within 60 days of completing their last therapy. This is the same group of patients who would be considered for Kyprolis-based therapy.
Moving forward, as ongoing clinical studies mature, we will start to see more use of Pomalyst and dexamethasone with other myeloma therapies.
The choice between Pomalyst and dexamethasone versus Kyprolis-based therapy should be dictated by response to prior therapy, tolerability of prior therapy, logistical issues (for example, travel back and forth to an infusion center), and cost. The great news is that patients who previously did not have good options now have two very good options from which to choose.
The last point is that Pomalyst is much more effective with the inclusion of dexamethasone, and there truly does appear to be clinical synergy between the two drugs in this combination. As such, Pomalyst should be used with once-weekly dexamethasone.
Frederic Reu, Cleveland Clinic:
I will use Pomalyst when I cannot control the patient's myeloma with another immunomodulatory drug and/or a proteasome inhibitor. This includes patients who either do not tolerate respective drugs or have a difficult time coming for treatment.
Edward Libby, Fred Hutchinson Cancer Research Center:
I will be using Pomalyst in patients in the later stages of myeloma: patients who have had several lines of therapy, many of whom will have relapsed after stem cell transplantation.
In all likelihood, I will combine Pomalyst with other agents whenever I can to enhance the response. At the recent ASH meeting, there were a number of reports on combining Pomalyst with steroids and Doxil (doxorubicin liposomal) or cyclophosphamide (Cytoxan).
The ease of administration of Pomalyst will be attractive to patients who live a significant distance from our cancer center.
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The Myeloma Beacon would like to thank the above physicians who participated in the survey for their assistance and expertise.
- Pomalyst (Pomalidomide) Approved By FDA For Relapsed And Refractory Multiple Myeloma
- Pomalyst Approved In Canada For Relapsed And Refractory Multiple Myeloma
- Pomalyst Plus Low-Dose Dexamethasone Is Active In High-Risk Relapsed Myeloma Patients (IMW 2013)
- Kyprolis Or Pomalyst For Dual-Refractory Myeloma - What Is The Survival Impact?
- Pomalyst-Based Combination Regimens Presented At ASH 2013