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FDA Panel Says ‘No’ to Multiple Myeloma Compound Panobinostat

Reviewers question risk–benefit profile

The FDA’s Oncologic Drugs Advisory Committee (ODAC) did not recommend approval of the investigational compound panobinostat (LBH589, Novartis), a pan-deacetylase (pan-DAC) inhibitor, for patients with previously treated multiple myeloma when used in combination with bortezomib and dexamethasone.

The vote was 5 to 2 against the drug.

According to documents released by the FDA before the vote, the question at hand was not whether the treatment was effective, but whether its risks outweighed its benefits.

The committee’s opinion will be considered by the FDA in its review of the new drug application for panobinostat, but the agency is not bound to follow its advisors’ guidance.

Data presented to the committee included the results from two clinical studies that evaluated panobinostat in combination with bortezomib and dexamethasone in patients with relapsed or relapsed/refractory multiple myeloma: a phase III randomized, double-blind, placebo-controlled, global registration trial (PANORAMA-1 [PANobinostat ORAl in Multiple MyelomA]) and a phase II U.S. single-arm, open-label study (PANORAMA-2).

Multiple myeloma is an incurable disease in which the normal epigenetic process is disrupted, resulting in the growth of cancerous plasma cells, potential resistance to treatment, and ultimately disease progression. It typically occurs in individuals 60 years of age or older, with few cases in individuals younger than 40.

If approved, panobinostat would be a first-in-class therapy for patients with previously treated multiple myeloma. As an epigenetic regulator, the drug may help restore the dysregulated cell programming that occurs in the disease.

Because panobinostat is an investigational compound, its safety and efficacy profiles have not been established.

Sources: Novartis; November 7, 2014; and BioSpace; November 6, 2014.

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