First-in-Class Drug Approved for Multiple Myeloma

First-in-Class Drug Approved for Multiple Myeloma
First-in-Class Drug Approved for Multiple Myeloma

The Food and Drug Administration (FDA) has approved Farydak (panobinostat; Novartis) for the treatment of patients with multiple myeloma who have received at least two prior therapies, including bortezomib and an immunomodulator. It is intended for use in combination with bortezomib and dexamethasone.

Farydak inhibits histone deacetylases (HDACs) that slows the over-development of plasma cells in multiple myeloma patients, or cause these cells to die. It is the first HDAC inhibitor approved for this condition.

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The safety and efficacy of Farydak in combination with bortezomib and dexamethasone was shown in a study (n=193) of patients with multiple myeloma who received at least two prior treatments that included bortezomib and an immunomodulator. Patients were randomized to receive a combination of Farydak, bortezomib, and dexamethasone, or bortezomib and dexamethasone alone. Study results showed that patients who received the Farydak combination had a progression-free survival for about 10.6 months vs. 5.8 months in patients who received bortezomib and dexamethasone. Also, over half (59%) of patients who received the Farydak combination had a response rate vs. 41% in patients who received bortezomib and dexamethasone.

Due to the risk of severe diarrhea, fatal cardiac events, arrhythmias, and electrocardiogram (ECG) changes, Farydak is being approved with a Risk Evaluation and Mitigation Strategy (REMS). Farydak was granted priority review and orphan product designation, and was reviewed under the FDA's accelerated approval program.

For more information visit FDA.gov.

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