FDA approves Farydak for multiple myeloma

The FDA today announced the approval of panobinostat for the treatment of multiple myeloma.

Panobinostat (Farydak, Novartis Pharmaceuticals) is the first histone deacetylase inhibitor designed to treat patients with multiple myeloma. Panobinostat slows the overdevelopment of plasma cells or causes the cancer cells to die and is intended to treat patients who have received at least two prior standard therapies, including bortezomib (Velcade, Millennium Pharmaceuticals) and dexamethasone.

“Farydak has a new mechanism of action that distinguishes it from prior drugs approved to treat multiple myeloma, making it a potentially attractive candidate agent for the treatment of multiple myeloma,” Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “Farydak’s approval is particularly important because it has been shown to slow the progression of multiple myeloma.”

The FDA based its decision on results of a study that analyzed data from 193 randomly assigned clinical trial participants who received either a combination of panobinostat, bortezomib and dexamethasone or bortezomib and dexamethasone alone.

Participants who received panobinostat in addition to bortezomib and dexamethasone demonstrated prolonged PFS (10.6 months) compared with participants treated with bortezomib and dexamethasone alone (5.8 months).

Response rate for participants in the panobinostat combination arm was 59% vs. 41% in the bortezomib and dexamethasone arm.

Patients who received panobinostat have experienced severe diarrhea, severe and fatal cardiac events, arrhythmias and electrocardiogram changes; therefore, the medication carries a boxed warning to alert patients and health care officials of these risks.

Panobinostat is being approved with a Risk Evaluation and Mitigation Strategy, which includes a communication plan to inform health care professionals of these risks and how to minimize them.

The most common adverse effects experienced by patients treated with panobinostat were diarrhea, tiredness, nausea, swelling in the arms or legs, decreased appetite, fever, vomiting and weakness.