Pomalidomid

Pomalidomide is an oral immunomodulatory imide drug (IMiD) used primarily in the treatment of multiple myeloma. It is a thalidomide analogue developed by Celgene and is marketed under the brand name Pomalyst, now distributed by Bristol Myers Squibb after the Celgene acquisition. As an IMiD, pomalidomide has anti-tumor, antiangiogenic, and immune-modulating effects.

Mechanism of action involves binding to cereblon, a component of the CRL4 E3 ubiquitin ligase complex, which leads to the ubiquitination and degradation of transcription factors IKZF1 (Ikaros) and IKZF3 (Aiolos). This modifies tumor cell survival and enhances immune activity, notably affecting T cells and natural killer cells. The drug is commonly used in combination with dexamethasone.

Clinical use and indications vary by country. In the United States, pomalidomide in combination with dexamethasone is approved for adults with relapsed or refractory multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor. It is typically employed after failure of other therapies, often as part of a multi-agent regimen or as a daily oral dose with cyclical dosing, with adjustments for cytopenias and comorbidities.

Safety and monitoring are important considerations. Adverse effects commonly include cytopenias (neutropenia, thrombocytopenia, anemia), fatigue, edema, diarrhea, and infections. Pomalidomide is teratogenic and is handled under strict pregnancy prevention programs in many jurisdictions; both male and female patients may be subject to restrictions, depending on local regulations. Thromboembolic risk is also a concern, and prophylaxis may be considered in some settings.

Regulatory status varies by region, but pomalidomide has been approved in multiple countries for relapsed or refractory multiple myeloma after at least two prior therapies.