What is Pomalyst (pomalidomide) given for?

Pomalyst (Pomalidomide) Treatment Indications

Pomalyst (pomalidomide) is FDA-approved for the treatment of multiple myeloma in patients who have received at least two prior therapies including lenalidomide and a proteasome inhibitor, and have demonstrated disease progression on or within 60 days of completion of the last therapy. 1

Primary Indications

Pomalidomide is indicated for:

1. Multiple Myeloma (MM):

   • Used in combination with dexamethasone
   • For patients who have received at least 2 prior therapies including:
     • Lenalidomide (an immunomodulatory drug/IMiD)
     • A proteasome inhibitor (PI) such as bortezomib
   • Disease must have progressed during treatment or within 60 days after completing the last therapy 2

2. AIDS-related Kaposi Sarcoma:

   • After failure of highly active antiretroviral therapy (HAART)
   • Or in HIV-negative patients with Kaposi Sarcoma 1

Efficacy in Multiple Myeloma

Clinical trials have demonstrated significant efficacy of pomalidomide plus dexamethasone in relapsed/refractory MM:

• In the pivotal MM-003 trial, pomalidomide plus low-dose dexamethasone showed:

  • Significantly longer progression-free survival (PFS) compared to high-dose dexamethasone (4 vs 1.9 months)
  • Improved overall survival (12.7 vs 8.1 months) 2

• Phase II studies showed overall response rates of 34-49% in heavily pretreated patients 2

• The MM-014 trial demonstrated an overall response rate of 32.1% and median PFS of 12.2 months in lenalidomide-refractory patients 3

Dosing and Administration

• FDA-recommended dose: 4 mg orally on days 1-21 of repeated 28-day cycles
• Typically combined with dexamethasone 40 mg weekly (20 mg for patients >75 years)
• Treatment continues until disease progression 2, 1

Combination Therapy Options

The NCCN Guidelines Panel has included several pomalidomide-based regimens:

• Pomalidomide plus dexamethasone (category 1 recommendation) 2
• Selinexor/pomalidomide/dexamethasone for patients with at least 2 prior therapies (category 1) 2

Common Adverse Effects

Hematologic:

• Neutropenia (most common dose-limiting toxicity)
• Thrombocytopenia
• Anemia 4, 5

Non-hematologic:

• Fatigue
• Pneumonia
• Risk of thromboembolic events 4, 5

Important Safety Considerations

• Pregnancy risk: Pomalidomide is a thalidomide analogue and can cause severe birth defects. A strict REMS program is required for prescribing and dispensing 1

• Thromboembolism risk: Blood clots in arteries, veins, and lungs can occur. Most patients will require anticoagulation prophylaxis 1

• Myelosuppression: Regular monitoring of complete blood counts is essential 4

• No dose adjustment is needed for moderate renal impairment, but close monitoring is recommended 4

Monitoring Requirements

• Pregnancy tests for females of reproductive potential
• Complete blood counts monthly
• Renal function assessment quarterly
• Regular assessment of myeloma disease status 4, 1

Pomalidomide represents an important treatment option for patients with multiple myeloma who have exhausted other therapies, particularly those who have become refractory to lenalidomide and bortezomib, offering meaningful improvements in survival outcomes despite advanced disease.