FDA advisers spurn Novartis' blood cancer drug, putting approval in doubt

Novartis' ($NVS) new multiple myeloma treatment is too risky to be approved, a group of FDA advisers voted, dealing a blow to the drugmaker and its hopes of launching the cancer drug next year.

The FDA's Oncologic Drugs Advisory Committee voted 5-2 against the drug, called panobinostat, noting its demonstrable benefits for patients with blood cancer but ultimately concluding that its side effects were too severe to warrant approval. In its pivotal trial, Novartis' treatment helped extend progression-free survival (PFS) by 3.9 months compared to placebo, but not without serious safety concerns. Seven percent of patients in the panobinostat arm died from non-cancer complications compared to just 3.5% in other group, alarming toxicity that outweighed the drug's benefit, panelists said.

The agency is not obligated to follow the committee's advice, though it most often does. Regulators are expected to hand down a final decision on the drug next month.

Bruno Strigini

"We are disappointed by this voting outcome and believe the results from our clinical trials provide strong evidence to support LBH589 as a potential first-in-class treatment option for multiple myeloma, a cancer where an unmet patient need exists," Novartis Oncology President Bruno Strigini said in a statement. "We will continue to work with the FDA as it completes its review of the U.S. application."

Tested in 768 patients with multiple myeloma, a combination of the panobinostat, Takeda's Velcade and the steroid dexamethasone significantly improved PFS, but the company's submission was rife with missing data, the committee pointed out. That's due, in part, to a high drop-out rate in the treatment arm, panelists said, as patients taking panobinostat reported symptoms including myelosuppression, hemorrhage, infection, gastrointestinal toxicity and cardiac toxicity.

The drug, which Novartis hopes to market as Farydak, works by blocking both histone and non-histone deacetylase enzymes--abbreviated as HDACs and DACs--putting serious stress on cancer cells until they die, all while leaving healthy cells unmarred. Novartis believes its "pan-DAC" inhibitor stands out among similar efforts from Celgene ($CELG), Acetylon Pharmaceuticals and MorphoSys.

Panobinostat is one of 10 pipeline treatments Novartis' ambitious oncology division plans to launch by 2017, and the Swiss drugmaker is on record with expectations to develop 14 blockbusters by 2018.

- read the statement

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