FDA approves multiple myeloma drug

February 23, 2015

FDA approved Farydak (panobinostat) for the treatment of patients with multiple myeloma, which is attributed to 10,710 deaths in the U.S. annually.

FDA approved panobinostat (Farydak, Novartis Pharmaceuticals) for the treatment of patients with multiple myeloma, which is attributed to 10,710 deaths in the United States annually.

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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.

Notably, 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, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research (CDER) in a statement. “Farydak’s approval is particularly important because it has been shown to slow the progression of multiple myeloma.”

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In November, 2014, FDA’s Oncologic Drugs Advisory Committee advised the agency that, based on the data reviewed, the Farydak’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 2 prior standard therapies, including bortezomib and an immunomodulatory agent.

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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 2 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%t of Farydak-treated participants saw their cancer shrink or disappear after treatment (response rate), versus 41% in those receiving bortezomib and dexamethasone.

Farydak carries a Boxed Warning, alerting patients and healthcare 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.

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.