Farydak - Panobinostat

What is Farydak - Panobinostat used for?

Farydak is a cancer medicine used in combination with two other medicines, bortezomib and dexamethasone, to treat multiple myeloma (a cancer of the bone marrow). It is administered to adults in whom the disease has reappeared or worsened after at least two previous treatments, including bortezomib and an immunomodulator (a medicine that acts on the immune system).

Farydak contains the active ingredient panobinostat.

Because the number of patients with multiple myeloma is low, the disease is considered 'rare' and Farydak was designated an 'orphan medicine' (a medicine used in rare diseases) on 8 November 2012.

How is Farydak - Panobinostat used?

Treatment with Farydak should be started by a doctor experienced in the use of anticancer therapies and the medicine can only be obtained with a prescription.

Farydak is available as capsules (10, 15 and 20 mg) and is given in 21-day treatment cycles, along with bortezomib and dexamethasone. The recommended starting dose of Farydak is 20 mg, taken on days 1, 3, 5, 8, 10 and 12 of the cycle. Patients receive the medicine for 8 cycles and a further 8 treatment cycles are recommended in patients who receive clinical benefit. Your doctor may change the dose or delay the administration in patients who experience severe side effects. For more information, see the summary of product characteristics (included with product information).

How does Farydak - Panobinostat work?

The active substance in Farydak, panobinostat, is a type of medicine called histone deacetylase (HDAC) inhibitor. It blocks the activity of enzymes called histone deacetylases (HDAC), involved in the activation and deactivation of genes within cells. In multiple myeloma, panobinostat is expected to keep genes that inhibit the division and growth of tumor cells activated. This is expected to stop the multiplication of c

What benefit has Farydak - Panobinostat shown during the studies?

The benefits of Farydak have been demonstrated in a main study conducted in 768 patients with multiple myeloma who had recurred after previous treatments. The medicine was compared with placebo (a dummy treatment) as an adjunct to bortezomib and dexamethasone treatment. The main measure of effectiveness was the mean time elapsed before the worsening of the patient's disease (progression-free survival), which was 12 months in patients treated with Farydak, compared to about 8 months in those treated with placebo.

When the results were analyzed only for the group of patients who had received at least two previous treatments, including bortezomib and an immunomodulatory medicine (thalidomide, lenalidomide or pomalidomide), the average time until myeloma worsened was 12.5 months with Farydak, compared to 4.7 months with placebo.

What is the risk associated with Farydak - Panobinostat?

The most common side effects with Farydak (which may affect more than 1 in 10 people) are diarrhea, fatigue, nausea and vomiting and blood-borne effects, such as thrombocytopenia (low levels of platelets, important for blood clotting), anemia and neutropenia and lymphopenia (low levels of some white blood cells). The most significant effects that led to the interruption of treatment in patients (occurring in about 4 patients out of 10) were diarrhea, weakness and fatigue and pneumonia (pulmonary infection). Effects on the heart occurred in 1-2 of 10 patients and included tachycardia (increased heart rate), palpitations and irregular heart rhythm (atrial fibrillation, sinus tachycardia); more rarely, patients experienced alterations in electrical conduction in the heart (prolongation of the QTc interval). For the full list of all side effects reported with Farydak, see the package leaflet.

Farydak should not be used in breast-feeding women. For the full list of limitations, see the package leaflet.

Why has Farydak - Panobinostat been approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) considered the increase in progression-free survival to be clinically significant, while noting that a benefit in terms of overall survival had not yet been demonstrated. Furthermore, panobinostat acts differently than existing treatments. This means that it offers a new alternative for patients subjected to at least two previous treatments, including bortezomib and immunomodulatory agents, which have limited therapeutic options and therefore a high unmet medical need. Although the undesirable effects caused concern and could not be justified in patients potentially treatable with less toxic therapies, the CHMP considered that they were acceptable in the subgroup treated above, given the absence of alternatives, and that they were manageable. Therefore, the Committee for Medicinal Products for Human Use (CHMP) decided that Farydak's benefits are greater than its risks in this group and recommended that it be approved for use in the EU.

What measures are being taken to ensure the safe and effective use of Farydak - Panobinostat?

A risk management plan has been developed to ensure that Farydak is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Farydak, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that markets Farydak will provide information material for patients, including a patient card, to help them take the medicine correctly. It will also provide a final analysis derived from the main study on the duration of survival of patients who have been treated with the medicine.