NOVARTIS: FARYDAK (panobinostat) FDA approved.for PLASMA CELL CANCER

• , 2015 03:18 PM EST

  Novartis receives FDA approval of Farydak®, the first HDAC inhibitor for patients with multiple myeloma

• ·  Farydak, an HDAC(HISTONE DEACETYLASE ) inhibitor with epigenetic activity, approved in combination for patients who received at least two prior regimens including bortezomib and IMiD1
  
  ·  Farydak prolonged median PFS benefit when used with bortezomib and dexamethasone combination versus combination alone (from 6 to 11 months)1
  
  ·  Multiple myeloma is an incurable blood cancer and there is an urgent need for new treatments2
  
  ·  Farydak is approved under FDA’s accelerated approval program; regulatory applications are underway in the EU, Japan and worldwide
  
  
  Basel, February 23, 2015 – Novartis announced today that the US Food and Drug Administration (FDA) has approved Farydak® (panobinostat, previously known as LBH589) capsules in combination with bortezomib* and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior regimens, including bortezomib and an immunomodulatory (IMiD) agent1.
  
  "Farydak represents an exciting agent with a new mechanism of action that is part of a promising class of drugs in this setting,” said study investigator Paul Richardson, MD, Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute. “Importantly, Farydak has been shown to improve progression-free survival in relapsed multiple myeloma patients who have received at least two prior regimens, including bortezomib and an IMiD, which is an area of particular unmet medical need.”
  
  Farydak has been shown to extend the progression-free survival (PFS) benefit of the standard-of-care therapy in this patient population1. Farydak is approved under accelerated approval based on PFS1. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. The FDA’s accelerated approval program gives patients access to treatments for serious or life-threatening illnesses that provide meaningful therapeutic benefit over existing treatments. The FDA has approved a risk evaluation and mitigation strategy (REMS) for Farydak. The REMS program serves to inform and educate healthcare professionals about the risks that may be associated with Farydak treatment.
  
  This FDA approval is based on efficacy and safety data in a pre-specified subgroup analysis of 193 patients who had received prior treatment with both bortezomib and an IMiD during the Phase III, randomized, double-blind, placebo-controlled, multicenter global registration trial, called PANORAMA-1 (PANobinostat ORAl in Multiple MyelomA)1. The trial found that the median PFS benefit increased in Farydak patients who had received prior treatment with both bortezomib and an IMiD (10.6 months; n=94), as compared to the placebo arm (5.8 months; n=99) (hazard ratio=0.52 [95% confidence interval (CI): 0.36, 0.76])1.
  
  The most common adverse reactions (incidence ≥ 20%) in clinical studies are diarrhea, fatigue, nausea, peripheral edema, decreased appetite, pyrexia and vomiting1. The most common non-hematologic laboratory abnormalities (incidence ≥ 40%) are hypophosphatemia, hypokalemia, hyponatremia and increased creatinine1. The most common hematologic laboratory abnormalities (incidence ≥ 60%) are thrombocytopenia, lymphopenia, leukopenia, neutropenia and anemia1. Farydak can cause fatal and serious toxicities including severe diarrhea and cardiac toxicities. Severe diarrhea occurred in 25% of Farydak-treated patients. Severe and fatal cardiac ischemic events, including severe arrhythmias and ECG changes have occurred in patients receiving Farydak. Serious adverse events (SAEs) occurred in 60% of patients treated with Farydak, bortezomib and dexamethasone compared to 42% of patients in the control arm. The most frequent (≥ 5%) treatment-emergent SAEs reported for patients treated with Farydak were pneumonia (18%), diarrhea (11%), thrombocytopenia (7%), fatigue (6%) and sepsis (6%). Additional serious adverse reactions included hemorrhage, myelosuppression, infections, hepatotoxicity and embryo-fetal toxicity1.
  
  “Novartis is committed to developing innovative first-in-class therapies for patients who need treatment options,” said Bruno Strigini, President, Novartis Oncology. “Farydak represents a new drug class in multiple myeloma, providing these patients with an important treatment approach for this difficult-to-treat cancer.”
  
  Farydak is the first histone deacetylase (HDAC) inhibitor available to patients with multiple myeloma3. As an HDAC inhibitor, its epigenetic activity may help to restore cell function in multiple myeloma4.
  
  Additional regulatory submissions for Farydak are being reviewed by health authorities worldwide.
  
  About multiple myeloma
  Epigenetics is the cell programming that governs gene expression and cell development3. In multiple myeloma, the normal epigenetic process is disrupted (also called epigenetic dysregulation) resulting in the growth of cancerous plasma cells, potential resistance to current treatment, and ultimately disease progression5,6.
  
  Multiple myeloma impacts approximately 1 to 5 in every 100,000 people globally7. Multiple myeloma is a cancer of the plasma cells, a kind of white blood cell present in bone marrow—the soft, blood-producing tissue that fills the center of most bones. The cancer is caused by the production and growth of abnormal cells within the plasma, which multiply and build up in the bone marrow, pushing out healthy cells and preventing them from functioning normally8. Multiple myeloma is an incurable disease with a high rate of relapse (when the cancer returns) and resistance (when the therapy stops working), despite currently available treatments2. It typically occurs in individuals 60 years of age or older, with few cases in individuals younger than 409.