Pomalidomide
Overview
Pomalidomide is an immunomodulatory anticancer molecule belonging to the class of thalidomide analogues, with both immune‑modulating and direct anti‑neoplastic properties. It interferes with multiple cellular processes, including modulation of cytokine production, enhancement of T‑cell and natural killer cell activity, inhibition of angiogenesis, and impairment of tumour cell proliferation. Pomalidomide is administered orally in capsule form and is used in specialized oncology settings, typically in adult patients with specific hematological malignancies. Due to its mechanism of action involving complex effects on the immune system and tumour microenvironment, pomalidomide has become a key therapy in the management of relapsed or refractory multiple myeloma and, under certain regulatory indications, Kaposi sarcoma. Clinical use is guided by disease characteristics, prior treatment history, and careful safety monitoring to balance therapeutic benefit against known risks of adverse reactions.
Background and Date of Approval
Pomalidomide was first approved by the United States Food and Drug Administration on February 8, 2013, under the brand name Pomalyst for the treatment of adult patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression. On May 14, 2020, the FDA granted accelerated approval to pomalidomide for treating adult patients with AIDS‑related Kaposi sarcoma after failure of highly active antiretroviral therapy or in HIV‑negative patients with Kaposi sarcoma, under accelerated approval based on overall response rate. Pomalidomide received marketing authorisation valid throughout the European Union on 5 August 2013 for treatment in combination with dexamethasone in adult patients with relapsed and refractory multiple myeloma. Additional generic pomalidomide products have been authorised by the European Medicines Agency with marketing authorisation dates in 2024, reflecting regulatory recognition of bioequivalence and therapeutic utility in oncology.
Uses
Pomalidomide is indicated for the treatment of adult patients with multiple myeloma in combination with dexamethasone after at least two prior therapies including lenalidomide and a proteasome inhibitor and disease progression on or within 60 days of completion of the last therapy. It is also used under accelerated regulatory indications for adult patients with AIDS‑related Kaposi sarcoma after failure of antiretroviral therapy and in certain HIV‑negative patients with Kaposi sarcoma. In Europe, generic pomalidomide products are authorised for use in multiple myeloma in combination with bortezomib and dexamethasone in adults who have received prior treatments including lenalidomide, and in combination with dexamethasone in patients with relapsed and refractory disease.
Administration
Pomalidomide is administered orally once daily, typically on days 1 through 21 of repeated 28‑day cycles, with or without food, as determined by the treating clinician. The starting dose is usually 4 milligrams per day for multiple myeloma, with adjustments made based on tolerability, hematologic parameters, and individual patient factors such as renal function. Treatment continues until disease progression or unacceptable toxicity. Dose modifications are common in response to adverse effects such as neutropenia or thrombocytopenia, and regular blood count monitoring is essential to guide safe dosing.
Side Effects
Commonly observed side effects during pomalidomide therapy include neutropenia, anemia, thrombocytopenia, fatigue, constipation, diarrhea, rash, peripheral edema, and dizziness. Gastrointestinal symptoms are frequent, and hematologic abnormalities are particularly notable due to the molecule’s impact on bone marrow function. The severity and frequency of these effects vary among patients, and most are managed with supportive care, dose adjustments, or temporary treatment interruptions under close medical supervision.
Warnings
Serious adverse events associated with pomalidomide include life‑threatening thrombocytopenia and neutropenia leading to infection or bleeding, peripheral neuropathy, severe rash including Stevens‑Johnson syndrome, and thromboembolic events such as deep vein thrombosis and pulmonary embolism. Pomalidomide carries a high risk of teratogenicity, and strict pregnancy prevention programmes are required for patients of child‑bearing potential. Treatment interruption or discontinuation should be considered in cases of severe toxicity.
Precautions
Prior to initiating pomalidomide, baseline assessments should include complete blood counts, liver and kidney function tests, and pregnancy status where applicable. Caution is required in patients with significant renal or hepatic impairment, and regular monitoring of hematologic parameters is essential during treatment. Concomitant use of strong CYP450 enzyme inducers or inhibitors may alter pomalidomide exposure and warrants clinical consideration. Due to its immunomodulatory effects, live vaccines may be less effective during treatment and should be administered according to clinical guidance outside of therapy periods.
Expert Tips
Patient selection for pomalidomide should focus on those with relapsed or refractory multiple myeloma meeting regulatory criteria and without contraindications to immunomodulatory therapy. Baseline and ongoing monitoring of blood counts, renal function, and peripheral neuropathy symptoms is essential to optimise safety. Educate patients on adherence to oral dosing schedules and prompt reporting of symptoms suggestive of infection, thromboembolism, or severe skin reactions. Counsel on the importance of pregnancy prevention measures and coordinate care with multidisciplinary oncology teams to integrate pomalidomide with other antimyeloma agents when indicated. Pharmacists should verify appropriate dosing adjustments, review concomitant medications for interactions, and provide guidance on managing common adverse effects through supportive care strategies.