Novartis AG's Farydak Wins FDA Approval After Setback

February 24, 2015
By Jessica Wilson, BioSpace.com Breaking News Staff

The U.S. Food and Drug Administration (FDA) announced on Monday that it has approved Novartis AG ’s multiple myeloma drug Farydak, also known as panobinostat. The FDA’s Oncologic Drugs Advisory Committee (ODAC recommended against the approval of Farydak in November 2014 because, in the committee’s opinion, the drug’s benefits did not outweigh its risks for patients with relapsed multiple myeloma. While the FDA is not bound to follow an advisory committee’s recommendation, the agency often does, so this reversal surprised many people.

A document released by the FDA prior to the November 2014 advisory committee meeting notes that that there was “an increased incidence of deaths not due to progressive disease (7 percent vs. 3.5 percent)” in patients who received the treatment combination with Farydak vs. those that did not, BioSpace reported in November.

In yesterday’s announcement, the FDA acknowledged the reversal and explained that after ODAC’s recommendation last fall, Novartis “submitted additional information supporting Farydak’s use for a different indication: patients with multiple myeloma who have received at least two prior standard therapies, including bortezomib and an immunomodulatory agent.”

The FDA noted that the safety and efficacy trials of Farydak involved 193 patients randomized 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 progression-free survival for about 10.6 months, compared to 5.8 months in participants treated with bortezomib and dexamethasone alone. In addition, 59 percent of Farydak-treated participants saw their cancer shrink or disappear after treatment, versus 41 percent in those receiving bortezomib and dexamethasone.

“Farydak’s approval is particularly important because it has been shown to slow the progression of multiple myeloma,” said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a statement. “Farydak has a new mechanism of action that distinguishes it from prior drugs approved to treat multiple myeloma.” Pazdur is referring to the fact that Farydak is the first histone deacetylase inhibitor (HDAC) approved to treat the disease.

Because of safety concerns, Farydak will carry a Boxed Warning to alert people that “severe diarrhea and severe and fatal cardiac events, arrhythmias and electrocardiogram (ECG) changes have occurred in patients receiving Farydak,” the FDA stated. Novartis must also develop a Risk Evaluation and Mitigation Strategy (REMS) to inform health care professions of these risks and how to minimize them.

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