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Panobinostat, a pan-DAC inhibitor, for relapsed or relapsed and refractory multiple myeloma


Multiple myeloma, a cancer of white blood cells predominantly affecting the bone marrow, impacts approximately 1 to 5 in every 100,000 people worldwide each year. With a five-year survival rate of 44%, there is an unmet treatment need for people living with this cancer.

Multiple myeloma typically occurs in individuals 50 years of age and older, with few cases in individuals younger than 40. Common symptoms include a high level of calcium in the blood, decreased red blood cells, kidney failure, bone damage and pain and fatigue, but may vary from person to person.

As a pan-DAC inhibitor, Panobinostat ( LBH589 ) potentially provides a novel mechanism of action to treat multiple myeloma and works by blocking a key class of cancer cell enzymes, which ultimately leads to cellular stress and death of these cells.

In May, Panobinostat was granted priority review by the FDA ( Food and Drug Administration ).

PANORAMA-1 data have shown that adding Panobinostat to the standard-of-care treatment for patients with relapsed or relapsed and refractory multiple myeloma offers an innovative and effective treatment option.

The PANORAMA-1 ( PANobinostat ORAl in Multiple MyelomA ) clinical trial is a phase III randomized, double-blind, placebo-controlled, multicenter global registration trial to evaluate Panobinostat in combination with Bortezomib ( Velcade ) and Dexamethasone against Bortezomib and Dexamethasone alone in patients with relapsed or relapsed and refractory multiple myeloma who failed on at least one prior treatment.
The study of 768 patients took place in 215 clinical trial sites worldwide.

The primary endpoint of the trial was progression-free survival. Data for overall survival, the key secondary endpoint of the trial, are not yet mature. Other secondary endpoints include overall response rate, duration of response and safety.

Panobinostat is a potent oral pan-inhibitor of class I, II, and IV histone ( and non-histone ) deacetylase enzymes ( HDACs/DACs ). ( Xagena )

Source: 50th Annual Meeting of the American Society of Clinical Oncology ( ASCO ), 2014

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