Relapsed/Refractory Patients:

Treatment Options - Pomalyst


Pomalyst®


NEWSFLASH: Pomalyst® (pomalidomide) now available for the treatment of relapsed and refractory multiple myeloma

On February 8, 2013 the FDA granted approval to Pomalyst® (pomalidomide) for patients who have received at least two prior therapies of established benefit which included both Velcade® (bortezomib) and Revlimid® (lenalidomide) and who have demonstrated disease progression on the last therapy. Click here to learn more about the role of the MMRF in advancing Pomalyst. Click here for more information from Celgene Corporation, the maker of Pomalyst.

To view full prescribing information, please visit: http://www.pomalyst.com/.

 

Overview

 

What is Pomalyst?

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

Pomalyst is also called pomalidomide and is 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) and Revlimid® (lenalidomide) and who have demonstrated disease progression on or within 60 days of completion of the last therapy.

The Multiple Myeloma Research Consortium (MMRC) has 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 does Pomalyst work?

Pomalyst works in multiple ways to slow or kill myeloma cells. It directly affects the tumor cells. It is also known 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 and stimulate the immune system.

How is Pomalyst and low-dose dexamethasone taken?

Pomalyst is taken orally at a dose of 4mg 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 40mg weekly. The dose of dexamethasone is reduced to 20mg weekly for patients older than 75 years old.

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

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

What are the possible side effects with Pomalyst and low-dose dexamethasone?

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 and fever associated with low white blood counts (febrile neutropenia)

  • Low red blood cell counts

  • Low platelets (thrombocytopenia)

  • Blood clots

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

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 called 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 types of patients can benefit from Pomalyst?

Pomalyst has been shown to be effective in patients with relapsed and refractory multiple myeloma following treatment with other available agents, including Velcade and Revlimid. In early studies 46% of patients who progressed after Velcade and Revlimid had clinical benefit from Pomalyst in combination with low-dose dexamethasone.

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

How effective is Pomalyst in treating relapsed and refractory multiple myeloma?

Pomalyst 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 were refractory to their last myeloma therapy and had received both Velcade and Revlimid.

  • This study showed that 29.2% of patients receiving the combination achieved a partial response or better.

  • The responses lasted a median of 7.4 months.

  • 7.4% of patients receiving Pomalyst alone achieved a partial response or better.

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 longer than those receiving high dose dexamethasone.

  • 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.

  • Overall responses rates were 24% with Pomalyst and low-dose dexamethasone versus 3% with high-dose dexamethasone alone.

 

How is Pomalyst currently being studied in myeloma?

Two Phase III studies are underway:

  • US study comparing Pomalyst plus Velcade-dexamethasone versus Velcade-dexamethasone in relapsed/refractory myeloma

  • International study being conducted outside the US evaluating of Pomalyst in patients with refractory or relapsed and refractory myeloma who were enrolled in an earlier Pomalyst study and discontinued treatment with high-dose dexamethasone due to disease progression

Other combinations are being evaluated in Phase I and Phase II studies including:

  • Phase II study in combination with Biaxin® (clarithromycin) and dexamethasone (ClaPd) in patients who have failed prior treatment with Revlimid +/- corticosteroids.

  • Phase I/II study in combination with Doxil® (pegylated liposomal doxorubicin) and dexamethasone

  • Phase I/II study in combination with high-dose dexamethasone and oral cyclophosphamide

  • In combination with Velcade and dexamethasone in two studies. A Phase I study that is being facilitated by the MMRC and a Phase I/II study.

  • Phase I study in combination with Kyprolis (carfilzomib) and dexamethasone that is being facilitated by the MMRC

Pomalyst monotherapy is being evaluated in Phase II studies in patients with relapsed/refractory myeloma.

  • Study comparing the effectiveness of continuous versus intermittent dosing

  • Study evaluating patients who have been determined to have high-risk myeloma based on gene expression profiling (GEP)

To find a clinical trial, call 1-866-603-MMCT (-6628) or click here to go to the MMRF Patient Navigator Program.



Reviewed by:
Keith Stewart, M.B.C.H.B.
Mayo Clinic
Scottsdale, AZ