What is Pomalyst?

Pomalyst® is an oral immunomodulatory agent (IMiD®) that is similar to Revlimid® (lenalidomide, Celgene) and Thalomid® (thalidomide, Celgene) but is more potent.

Pomalyst is also called pomalidomide and is also made by Celgene. In the United States, Pomalyst is approved for the use in patients with myeloma who have received at least two prior therapies, including Velcade® (bortezomib, Takeda Oncology) or another proteasome inhibitor and Revlimid and who have demonstrated disease progression on or within 60 days of completion of the last therapy.

The Multiple Myeloma Research Consortium (MMRC) played a substantial role in the development of this drug as a partner with Celgene in the early clinical trials establishing its efficacy and continues to sponsor trials evaluating Pomalyst in combination with various myeloma drugs.

How is Pomalyst used?

Pomalyst® (pomalidomide, Celgene) works in multiple ways to slow or kill myeloma cells. Recently it has been shown in studies the MMRC helped to fund that Pomalyst and other IMiDs bind a protein in myeloma cells called cereblon. This binding triggers myeloma cell death. In some studies higher levels of cereblon are associated with better outcomes after treatment. Pomalyst is also known to stimulate the immune system and to affect the blood vessels and other substances surrounding a tumor (known as the tumor microenvironment) which help to feed the cancer cell’s growth.

What types of patients can benefit from Pomalyst?

Pomalyst has been shown to be effective in patients with relapsed and refractory multiple myeloma following multiple lines of treatment with other available agents, including Velcade and Revlimid.

In addition, Pomalyst is effective in patients with high-risk myeloma as well as those with kidney problems.

How is Pomalyst administered?

Pomalyst® (pomalidomide, Celgene) is an oral medication that is taken along with dexamethasone. The recommended starting dose of Pomalyst is 4 mg once daily for three out of four weeks (days 1-21 of each 28-day cycle). Each Pomalyst capsule should be taken with water. Pomalyst should not be taken with food and should be taken at least 2 hours before or 2 hours after a meal. Dexamethasone is taken at a dose of 40 mg weekly, which is also referred to as “low-dose dexamethasone.” The dose of dexamethasone is reduced to 20 mg weekly for patients older than 75 years of age.

Typically, aspirin or another blood thinner is taken along with Pomalyst and low-dose dexamethasone.

Pomalyst and low-dose dexamethasone are taken for as long as they continue to work against the myeloma.

What are Pomalyst side effects?

Pomalyst® (pomalidomide, Celgene) side effects primarily depend on individual’s past health history and stage of their myeloma. Pomalyst and low-dose dexamethasone is generally well-tolerated. The most common side effects seen in the Phase II study upon which approval was granted were:

  • Fatigue and weakness
  • Low white blood cell counts (neutropenia)
  • Low red blood cell counts (anemia)
  • Gastrointestinal effects (constipation, nausea, or diarrhea)
  • Shortness of breath
  • Upper respiratory infection
  • Back pain
  • Fever

The most common serious side effects seen in the Phase II study were:

  • Low white blood cell counts
  • Pneumonia
  • Low red blood cell counts
  • Low platelets (thrombocytopenia)
  • Fatigue and weakness

A similar side effect profile was seen in a Phase III study that evaluated Pomalyst and low-dose dexamethasone.

If you experience low white blood cell or platelet counts or other serious side effects while receiving Pomalyst, your doctor may adjust your dose.

Some patients who received Pomalyst in clinical trials developed blood clots. For this reason, you will also receive aspirin or another blood thinner along with Pomalyst.

Women who are pregnant or who plan to become pregnant must not take Pomalyst. This precaution is due to its similarity to thalidomide, and some signs of birth defects in animals. A program has been created (Pomalyst REMS™) to prevent exposure to Pomalyst during pregnancy.

  • Doctors and pharmacists must register with the program in order to prescribe and dispense Pomalyst.
  • Patients must register and meet all the conditions of the program in order to take Pomalyst.

What have Pomalyst clinical studies shown?

Pomalyst® (pomalidomide, Celgene) was granted approval by the US Food and Drug Administration based on the results of a Phase II trial that evaluated Pomalyst alone and in combination with low-dose dexamethasone. The study included 221 patients with relapsed myeloma who had received an average of 5 previous therapies, were refractory to their last myeloma therapy, and had received both Velcade® (bortezomib, Takeda Oncology) and Revlimid® (lenalidomide, Celgene).

  • This study showed that 33% of patients receiving the combination and 18% of patients receiving Pomalyst alone achieved a partial response or better.
  • The responses lasted a median of 8.3 months in the combination group and 10.7 months in the Pomalyst group.
  • On average, the overall survival was 16.5 months in the combination group and 13.6 months in the Pomalyst group. This is longer than what has historically been seen in patients in whom currently approved novel therapies have failed.
  • Pomalyst-low-dose-dexamethasone was just as effective in patients who had received Revlimid as part of their last treatment and whose myeloma no longer responded to Revlimid or both Revlimid and Velcade.

The combination of Pomalyst and low-dose dexamethasone was compared to high-dose dexamethasone in a Phase III trial with 455 patients with refractory myeloma who had failed therapy with both Velcade and Revlimid, given either alone or in combination.

  • This study showed that patients receiving the combination of Pomalyst plus low-dose dexamethasone lived significantly longer than those receiving high dose dexamethasone (12.4 months versus 8 months).
  • Overall responses rates were 24% with Pomalyst and low-dose dexamethasone versus 4% with high-dose dexamethasone alone.
  • On average, time without progression of myeloma was 3.6 months with Pomalyst and low-dose dexamethasone versus 1.8 months with high-dose dexamethasone alone.
  • The combination was effective in patients with high-risk myeloma and those who previously received Revlimid.

Pomalyst-low-dose dexamethasone is listed as one of several preferred regimens for previously treated myeloma in current treatment guidelines.

Results of several early-stage trials have evaluated the addition of a third agent to Pomalyst-dex in relapsed and/or refractory myeloma. For example:

  • Results of a randomized Phase II trial that enrolled 80 patients who no longer responded to Revlimid showed that adding cyclophosphamide improved the overall response rate and time without progression over that seen with Pomalyst-low-dose dex.
  • Preliminary results of a small Phase II trial of the combination of Pomalyst-low-dose dex and Keytruda® (pembrolizumab, Merck), a type of immunotherapy known as a checkpoint inhibitor, showed early evidence of deep, durable responses, including in patients with high-risk disease.
  • Preliminary results of an ongoing multicenter Phase 1b study showed that the combination of Darzalex and Pomalyst-dex resulted in a high 71% overall response rate in heavily pretreated patients.

How is Pomalyst currently being studied in myeloma?

Pomalyst is being evaluated in a number of ongoing clinical trials in relapsed and/or refractory myeloma, including two Phase III trials.

  • The combination of Pomalyst, Velcade, and low-dose dexamethasone is being compared to Velcade-low-dose dex in relapsed or refractory myeloma.
  • Pomalyst-low-dose dex is being evaluated in combination with Keytruda in relapsed and/or refractory myeloma.

Interested in learning more about Pomalyst clinical trials? Talk to an MMRF Patient Support Nurse to learn more or use our clinical trials finder.