The FDA today approved Novartis Pharmaceuticals' panobinostat (Farydak) for the treatment of patients with multiple myeloma who have received at least 2 prior standard therapies.
The FDA today approved Novartis Pharmaceuticals’ panobinostat (Farydak), a histone deacetylase (HDAC) inhibitor, for the treatment of patients with multiple myeloma who have received at least 2 prior standard therapies, including bortezomib and an immunomodulatory agent.
Prior to Farydak’s approval, the FDA had designated the drug as an orphan product and granted it priority review status.
The FDA based its nod on the results of a clinical trial evaluating the safety and efficacy of Farydak in combination with bortezomib and dexamethasone in 193 multiple myeloma patients. The research team found that patients treated with Farydak in combination with bortezomib and dexamethasone experienced an average 10.6 month delay in disease progression; comparatively, patients receiving bortezomib and dexamethasone alone experienced an average disease progression delay of 5.8 months.
The researchers also discovered cancer reduction or disappearance in 59% of Farydak-treated participants, compared with 41% of patients treated with bortezomib 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,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “Farydak’s approval is particularly important because it has been shown to slow the progression of multiple myeloma.”
The most common adverse events experienced by trial participants treated with Farydak included diarrhea, tiredness, nausea, swelling in the arms or legs, decreased appetite, fever, vomiting and weakness, hypophosphatemia, hypokalemia, hyponatremia, increased creatinine, thrombocytopenia, leukopenia, and anemia. The use of Farydak is also associated with hepatotoxicity, severe diarrhea, arrhythmias and electrocardiogram changes, and severe and fatal cardiac events.
In November 2014, the FDA’s Oncologic Drugs Advisory Committee advised against the approval of Farydak for patients with relapsed multiple myeloma, stating that the data reviewed did not indicate the that the drug’s potential benefits outweighed its risks. Following this recommendation, Novartis presented additional data supporting the use of Farydak for patients with multiple myeloma who have received at least 2 prior standard therapies, including bortezomib and an immunomodulatory agent.
Approximately 21,700 Americans are diagnosed with multiple myeloma and 10,710 die from the disease every year, according to the National Cancer Institute.