Pomalyst®

What is Pomalyst?

Pomalyst, also known as pomalidomide, is an oral immunomodulatory agent (IMiD) that is similar to Revlimid (lenalidomide, Celgene) and Thalomid (thalidomide, Celgene) but is more potent. Pomalyst is manufactured by Celgene.

The Multiple Myeloma Research Consortium (MMRC) played an integral role in the development of Pomalyst, partnering with Celgene in the early clinical trials that established its efficacy, and continues to sponsor trials evaluating Pomalyst in combination with other myeloma drugs.


How is Pomalyst used?

n the U.S., Pomalyst  is approved for use in patients with myeloma who have received at least two prior therapies, including Revlimid and a proteasome inhibitor, and have demonstrated disease progression on or within 60 days of completion of the last therapy.

What types of patients can benefit from Pomalyst?

Pomalyst has been shown to be effective in patients with relapsed and refractory multiple myeloma who have received multiple lines of treatment with other agents, including Velcade (bortezomib, Takeda Oncology) and Revlimid.

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

 

How does Pomalyst work?

Pomalyst works in multiple ways to slow or kill myeloma cells. Studies funded by the MMRC showed that Pomalyst and other IMiDs bind a protein in myeloma cells called cereblon, which triggers myeloma cell death. In some studies, higher levels of cereblon were 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) that help to feed the cancer cell’s growth.


How is Pomalyst administered?

Pomalyst 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 121 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. 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?

The most common side effects of Pomalyst (with or without dexamethasone) that were seen in phase 2 and 3 studies include:

  • Fatigue and weakness
  • Low counts of red blood cells (anemia), white blood cells (neutropenia), or platelets (thrombocytopenia)
  • Swelling of the hands or feet (peripheral edema)
  • Pain, numbness, or tingling in the hands and feet (peripheral neuropathy)
  • Gastrointestinal effects (constipation, nausea, or diarrhea)
  • Shortness of breath
  • Upper respiratory infection or cough
  • Back pain
  • Musculoskeletal pain
  • Muscle spasms
  • Fever
  • Pneumonia
  • Decreased appetite
  • Dizziness
  • Rash

The most common serious side effects that were seen in phase 2 and 3 studies include:

  • Low counts of red blood cells (anemia), white blood cells (neutropenia), or platelets (thrombocytopenia)
  • Fever from a low white blood cell count (febrile neutropenia)
  • Pneumonia
  • Fatigue and weakness
  • Muscle weakness
  • Urinary tract infection
  • Blood infection
  • High blood calcium
  • Shortness of breath
  • Increased blood creatinine
  • Confusion
  • Renal failure
  • Back pain or bone pain

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. The Pomalyst Risk Evaluation and Mitigation Strategies (REMS) program was created 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.

How are side effects of Pomalyst managed?

Patients who experience low white blood cell or platelet counts or other serious side effects while receiving Pomalyst may have their dose adjusted.

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


What have Pomalyst clinical studies shown?

Pomalyst was granted approval by the US Food and Drug Administration based on the results of a phase 2 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 five previous therapies, were refractory to their last myeloma therapy, and had received both Velcade and Revlimid .

  • 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 7.4 months in the combination group and was not yet established in the Pomalyst group.

The combination of Pomalyst and low-dose dexamethasone was compared to high-dose dexamethasone in a phase 3 trial with 455 patients with refractory myeloma who had received at least two prior treatment regimens, including Velcade and Revlimid, and demonstrated disease progression within 60 days of their last therapy.

  • 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 vs 8 months, respectively).
  • 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 vs 1.8 months with high-dose dexamethasone alone.
  • The combination was effective in patients with high-risk myeloma and those who previously received Revlimid.

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 three phase 3 trials.

  • The combination of Pomalyst, Velcade, and low-dose dexamethasone is being compared to Velcadelow-dose dexamethasone in relapsed or refractory myeloma. (OPTIMISMM trial; NCT01734928)
  • Pomalystlow-dose dexamethasone is being evaluated in combination with Keytruda (pembrolizumab, Merck) in relapsed and/or refractory myeloma. (KEYNOTE-183 trial; NCT02576977)
  • Pomalyst-dexamethasone is being evaluated in combination with Opdivo (nivolumab, Bristol-Myers Squibb) with or without Empliciti (elotuzumab, Bristol-Myers Squibb). (CheckMate 602 trial; NCT02726581

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

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 Nurse Patient Navigator to learn more or use our clinical trials finder.

FIND CLINICAL TRIALS

Updated April 2019

Please visit the sponsor’s official patient site for up-to-date information on Pomalyst.