Silver Spring, MD (Press Release) - The U.S. Food and Drug Admin­istra­tion today approved Farydak (panobinostat) for the treat­ment of patients with multiple myeloma.

Multiple myeloma is a form of blood cancer that arises from plasma cells, a type of white blood cell, found in bone marrow. According to the National Cancer Institute, approx­i­mately 21,700 Americans are diag­nosed with multiple myeloma and 10,710 die from the disease annually.

Primarily affecting older adults, multiple myeloma causes plasma cells to rapidly multiply and crowd out other healthy blood cells from the bone marrow. When the bone marrow has too many plasma cells, the cells may move to other parts of the body, which can weaken the body’s immune system, lead to anemia and cause other bone and kidney problems.

Farydak works by inhibiting the activity of enzymes, known as histone deacetylases (HDACs). This process 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 approved to treat multiple myeloma. It 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.

“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,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Farydak’s approval is particularly important because it has been shown to slow the pro­gres­sion of multiple myeloma.”

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 saw 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).

The FDA granted Farydak priority review and orphan prod­uct desig­na­tion. Priority review provides for an expedited review of drugs that are intended to treat a serious disease or con­di­tion and may provide a sig­nif­i­cant im­prove­ment over avail­able ther­apy. Orphan prod­uct desig­na­tion is given to drugs intended to treat rare diseases.

The FDA action was taken under the agency’s accelerated approval pro­gram, which allows approval of a drug to treat a serious or life-threatening disease based on clin­i­cal data showing the drug has an effect on a surrogate end­point reason­ably likely to predict clin­i­cal benefit to patients. The accelerated approval pro­gram provides earlier patient access to promising new drugs while the com­pany conducts con­firmatory clin­i­cal trials. An im­prove­ment in survival or disease-related symp­toms has not yet been estab­lish­ed for Farydak. The com­pany is now required to conduct con­firmatory trials to verify and describe the clin­i­cal benefit of Farydak.

Farydak is marketed by East Hanover, New Jersey-based Novartis Pharma­ceu­ticals.

The FDA, an agency within the U.S. Department of Health and Human Services, promotes and protects the public health by, among other things, assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological prod­ucts for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supple­ments, prod­ucts that give off electronic radiation, and for regulating tobacco prod­ucts.

Source: Food and Drug Admin­istra­tion.