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Panobinostat First HDAC Inhibitor Approved by the FDA for the Treatment of Patients with Multiple Myeloma

Value-Based Care in Myeloma - FDA News & Updates, Multiple Myeloma

On February 23, 2015, the FDA approved panobinostat (Farydak; Novartis Pharmaceuticals) for the treatment of patients with multiple myeloma. Panobinostat works by inhibiting the histone deacetylase (HDAC) enzymes, which slow and/or kill the excess production of plasma cells in the bone marrow that leads to the development of multiple myeloma. Panobinostat was approved under the FDA’s priority review and accelerated approval programs and carries an orphan drug designation.

Panobinostat is the first HDAC inhibitor approved by the FDA for the treatment of patients with myeloma. It is indicated to be used in patients who have received at least 2 previous standard myeloma therapies, including bortezomib and an immunomodulatory agent (eg, lenalidomide). Panobinostat should be used in combination with bortezomib and dexamethasone.

The clinical trial that led to the approval of panobinostat involved 193 patients with myeloma who had received at least 2 previous treatments, including bortezomib and an immunomodulatory agent. In the trial, the combination of panobinostat, bortezomib, and dexamethasone slowed the progression of myeloma, resulting in an extended progression-free survival (PFS) by 4.10 months compared with bortezomib and dexamethasone alone. The PFS was approximately 10.6 months with the addition of panobinostat to the treatment regimen versus 5.8 months with bortezomib and dexamethasone alone. In addition, the response rate was 59% among the patients receiving the 3-drug combination versus 41% among those receiving bortezomib and dexamethasone without panobinostat.

The most common side effects seen with panobinostat were diarrhea, tiredness, nausea, swelling in the arms or legs, decreased appetite, fever, vomiting, and weakness. The most common laboratory abnormalities were hypophosphatemia, hypokalemia, hyponatremia, increased creatinine, thrombocytopenia, leukopenia, and anemia. Panobinostat carries a boxed warning about the risk for severe diarrhea and severe and fatal cardiac events. Because of these risks, the FDA requires a Risk Evaluation and Mitigation Strategy program to inform providers of these risks and how to minimize them.

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Last modified: May 20, 2015
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