FDA Approves Drug for Multiple Myeloma

The U.S. Food and Drug Administration has approved Farydak (panobinostat) to treat patients diagnosed with multiple myeloma.

This form of blood cancer, found in white blood cells known as plasma cells is diagnosed in about 21,700 people annually, according to the National Cancer Institute. About 10,710 people die from the disease each year. It is typically only diagnosed in older adults.

Drug is first of its kind to be approved for treating multiple myeloma

Farydak is a histone deacetylases (HDACs) inhibitor and the first drug of this kind to be approved for this cancer. It is intended for patients who have received at least two prior standard therapies, including bortezomib (Velcade) and an immunomodulatory agent such as thalidomide or Revlimid. Farydak is to be used in combination with bortezomib and dexamethasone.

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

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

Study shows Fardyk almost doubles progresion-free survival rates

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 two 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 that those patients in the study group receiving the Farydak combination experienced a progression-free survival of around 10.6 months compared to 5.8 months in those patients receiving bortezomib and dexamethasone.

Additionally, 59 percent of those in the study group responded to the treatment whereas only 41 percent of the other group responded to treatment.

Farydak carries a Boxed Warning alerting patients and health care 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.

Source: FDA