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FDA Postpones Review of Multiple Myeloma Compound Panobinostat

Decision follows ‘thumbs down’ from advisory panel

The FDA has extended its priority review period by up to 3 months for the new drug application (NDA) for panobinostat (LBH589, Novartis) in combination with bortezomib (Velcade, Millennium Pharmaceuticals) and dexamethasone for patients with previously treated multiple myeloma.

The NDA for panobinostat was submitted in March 2014. In May, the agency granted priority review status to the compound, reducing the standard 12-month review period to 8 months.

The current extension to the panobinostat NDA review period followed an FDA Oncologic Drugs Advisory Committee (ODAC) meeting earlier this month. At that time, the panel voted 5 to 2 against recommending the drug’s approval, concluding that its adverse effects were too severe to warrant approval. Agency reviewers are not beholden to the opinions of their independent panels, but they tend to side with the experts, putting eventual approval of the drug in jeopardy.

Multiple myeloma is an incurable cancer of plasma cells with a high rate of relapse, and patients often become refractory, despite currently available treatments. The disease typically occurs in individuals 60 years of age or older, with few cases in individuals younger than 40.

Epigenetics is the cell programming that governs gene expression and cell development. In multiple myeloma, the normal epigenetic process is disrupted (epigenetic dysregulation), resulting in the growth of cancerous plasma cells, potential resistance to current treatment, and ultimately disease progression.

Panobinostat is a potent pan-deacetylase (pan-DAC) inhibitor that, if approved, could be a first-in-class treatment for patients with relapsed or relapsed/refractory multiple myeloma. As an epigenetic regulator, panobinostat may help restore cell programming.

Because panobinostat is an investigational compound, its safety and efficacy profiles have not been established. Access to the compound is available only through carefully controlled and monitored clinical trials.

Sources: Novartis; November 25, 2014; and FierceBiotech; November 25, 2014.

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