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Farydak (Panobinostat) Gets FDA Nod for Treatment of Multiple Myeloma

Agency requires confirmatory trials to verify drug’s clinical benefits

The FDA has approved Farydak (panobinostat, Novartis) for the treatment of patients with multiple myeloma (MM).

According to the National Cancer Institute, approximately 21,700 Americans are diagnosed with MM and 10,710 die from the disease annually. MM primarily affects older adults.

Panobinostat works by inhibiting the activity of enzymes known as histone deacetylases (HDACs), which may slow the over-development of plasma cells or may cause these cells to die in patients with MM.

Panobinostat is the first HDAC inhibitor approved to treat MM. It is intended for patients who have received at least two prior standard therapies, including bortezomib and an immunomodulatory agent. Panobinostat 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 MM. After the meeting, the company submitted additional information supporting the use of panobinostat for a different indication: patients with MM who have received at least two prior standard therapies, including bortezomib and an immunomodulatory agent.

The safety and efficacy of panobinostat in combination with bortezomib and dexamethasone was demonstrated in 193 clinical trial participants with MM who received at least two prior treatments that included bortezomib and an immunomodulatory agent. The subjects were randomly assigned to receive a combination of panobinostat, bortezomib, and dexamethasone, or bortezomib and dexamethasone alone.

The results showed that participants receiving the panobinostat combination experienced a delay in their disease progression (progression-free survival) for approximately 10.6 months compared with 5.8 months among the subjects treated with bortezomib and dexamethasone alone. In addition, 59% of panobinostat-treated participants saw their cancer shrink or disappear after treatment (response rate) compared with 41% of those receiving bortezomib and dexamethasone.

The labeling for Farydak (panobinostat) includes a boxed warning alerting health care professionals and patients that severe diarrhea and severe and fatal cardiac events, arrhythmias, and electrocardiogram changes have occurred in patients receiving panobinostat. Because of these risks, panobinostat was 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 adverse effects associated with panobinostat included diarrhea, tiredness, nausea, swelling in the arms or legs, decreased appetite, fever, vomiting, and weakness. The most common laboratory abnormalities included hypophosphatemia, hypokalemia, hyponatremia, increased creatinine levels, thrombocytopenia, leukopenia, and anemia. Health care professionals should also inform patients of the risk of bleeding in the gastrointestinal tract and the lungs, and the risk of hepatotoxicity.

An improvement in survival or disease-related symptoms has not yet been established for panobinostat. Novartis is now required to conduct confirmatory trials to verify and describe the drug’s clinical benefits.

Source: FDA; February 23, 2015.

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