New blood cancer drug gets FDA approval23 Feb 2015
The U.S. FDA approved Novartis' drug, Farydak, to treat patients with multiple myeloma. The eligible patients must have relapsed after earlier therapies for multiple myeloma. Although in November, an advisory panel recommended against the drug, the FDA granted approval earlier this week.
Farydak was approved for use in combination with Takeda’s Velcade and the anti-inflammatory drug dexamethasone once a patient has received at least 2 prior treatment regimens. These prior treatments would include Velcade and an immunomodulatory drug, such as Celgene’s Revlimid.
Positive outcomes combined with serious side effects
The drug, Farydak, in clinical trials almost doubled the amount of time it took for the disease to progress, to 10.6 months compared with standard treatment. But it was associated with a wide array of serious side effects, including severe diarrhea and heart problems, which are prominently listed in a boxed warning.
The drug got approved even though the FDA's advisory panel of outside medical experts had voted 5 to 2 against recommending approval, saying the drug's benefits did not outweigh its risks.
Conditional approval and a special action plan
The FDA usually follows the advice of advisory panels even though it is obligated to do so. In this case, Farydak was granted conditional approval, which means that continued approval may hinge on demonstrating benefits in confirmatory trials.
Moreover, Farydak was approved with a Risk Evaluation and Mitigation Strategy (REMS), a communication plan meant to inform health care professionals of a specific drug's risks and how to minimize them.
The Novartis medicine is the first among a new class of treatments called HDAC inhibitors, meant to help restore the cell function and control the overgrowth of plasma cells, which causes the disease.