Credit: Steven Harbour
The US Food and Drug Administration (FDA) has granted accelerated approval for the immunomodulatory agent pomalidomide (Pomalyst) to treat patients with advanced multiple myeloma (MM).
Continued FDA approval for the drug may be contingent upon verification and description of clinical benefit in confirmatory trials.
Pomalidomide is intended for use in combination with dexamethasone to treat MM patients who have received at least 2 prior
therapies (including lenalidomide and a proteasome inhibitor) and who experienced progression within 60 days of their last treatment.
Pomalidomide has demonstrated some efficacy in this patient population in a number of studies.
In a study published in Blood last year (PG Richardson et al.), pomalidomide elicited responses in MM patients who were refractory to lenalidomide, bortezomib, or both drugs.
In a study presented at ASH 2011 (abstract 634), pomalidomide did not fare as well when given alone to patients with refractory MM. However, combining the drug with low-dose dexamethasone significantly improved responses.
A study presented at ASH 2012 (LBA-6) built upon those findings, showing that pomalidomide plus low-dose dexamethasone was superior to high-dose dexamethasone in MM patients who were refractory to lenalidomide and bortezomib.
Common side effects observed with pomalidomide include neutropenia, anemia, thrombocytopenia, fatigue, weakness, constipation, diarrhea, upper respiratory tract infections, back pain, and fever.
In addition, pomalidomide has been shown to cause venous thromboembolism, as well as severe, life-threatening birth defects in pregnant women. The drug carries a boxed warning alerting patients and healthcare professionals to both of these risks.
Because of the embryo-fetal risk, pomalidomide is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the program by enrolling and complying with the REMS requirements.
Patients must sign a patient-physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements.
Pharmacies must be certified with the Pomalyst REMS Program, must only dispense the drug to patients who are authorized to receive it, and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS.
Pomalidomide is marketed by Celgene, which is based in Summit, New Jersey.
