The FDA today approve Farydak (panobinostat) for the treatment of patients with multiple myeloma, a form of blood cancer arising from plasma cells, a type of white blood cell, found in bone marrow.
Multiple myeloma primarily affects older adults, causing plasma cells to rapidly multiple and crowd our other healthy blood cells from the bone marrow. When the bone marrow has too many plasma cells, they being to move to other parts of the body, which can then weaken the body’s immune system, leading to anemia and cause other bone and kidney problems.
Farydak works by inhibiting the activity of enzymes, known as histone deacetylases (HADCs); a process which may slow the over-development of plasma cells in multiple myeloma patients, or cause these dangerous cells to die.
Farydak is the first HDAC inhibitor approve to treat multiple myeloma, and is intended for patients who have received at least two prior standard therapies including bortezomib and an immunomodulatory agent. Farydak is to be used in combination with bortezomib, a type of chemotherapy, and dexamethasone, an anti-inflammatory medication.
In November 2014, the FDA’s Oncologic Drugs Advisory Committee advised the agency that, based on the data reviewed, the drug’s benefits did not outweigh its risks for patients with relapsed multiple myeloma. After the meeting, the company submitted additional information supporting Farydak’s use for a different indication: patients with multiple myeloma who have received at least two prior standard therapies, including bortezomib and an immunomodulatory agent.
The safety and efficacy of Farydak in combination with bortezomib and dexamethasone was demonstrated in 193 clinical trial participants with multiple myeloma who received at least two prior treatments that included bortezomib and an immunomodulatory agent. Participants were randomly assigned to receive a combination of Farydak, bortezomib and dexamethasone, or bortezomib and dexamethasone alone.
Study results showed participants receiving the Farydak combination experienced a delay in their disease progression (progression-free survival) for about 10.6 months, compared to 5.8 months in participants treated with bortezomib and dexamethasone alone. Additionally, 59 percent of Farydak-treated participants saw their cancer shrink or disappear after treatment (response rate), versus 41 percent in those receiving bortezomib and dexamethasone.
Farydak carries a Boxed Warning alerting patients and health care professionals that severe diarrhea and severe and fatal cardiac events, arrhythmias and electrocardiogram (ECG) changes have occurred in patients receiving Farydak. Because of these risks, Farydak is being approved with a Risk Evaluation and Mitigation Strategy (REMS) consisting of a communication plan to inform health care professionals of these risks and how to minimize them.
The most common side effects of Farydak were diarrhea, tiredness, nausea, swelling in the arms or legs, decreased appetite, fever, vomiting and weakness. The most common laboratory abnormalities were low levels of phosphorus in the blood (hypophosphatemia), low potassium levels in the blood (hypokalemia), low levels of salt in the blood (hyponatremia), increased creatinine, low platelets (thrombocytopenia), low white blood cell counts (leukopenia) and low red blood cell counts (anemia). Healthcare professionals should also inform patients of the risk of bleeding in the gastrointestinal tract and the lungs, and liver damage (hepatotoxicity).
Farydak is marketed by East Hanover, New Jersey-based Novartis Pharmaceuticals.