FDA Advisors Reject Novartis’ Multiple Myeloma Drug

The FDA oncology-drug advisory committee turned down Novartis’ investigational multiple myeloma drug Farydak by a 5-2 vote, citing safety concerns that largely overwhelmed benefits.

Based on studies evaluating Farydak (panobinostat) taken with Velcade (bortezomib) and dexamethasone in 768 patients with relapsed or treatment-resistant disease, the drug had a median progression-free survival (PFS) benefit of 3.9 months.

But this figure may be inaccurate, as Novartis excluded many patients from its analysis, said James Liebmann, M.D., a professor of medicine at the University of Massachusetts and one of the “no” votes.

Even given the PFS benefit, patients in the drug arm of the study showed an excess number of deaths over those in the control arm, he added. Roughly 30 patients, or 7.9 percent, in the study arm died, compared with 18 patients, or 4.8 percent, in the placebo arm. Most of those patients either died of disease progression or adverse events such as infection and hemorrhaging, Novartis said in its briefing materials.

While greater toxicity is expected when adding a third drug to the Velcade/dexamethasone regimen, one has to weigh whether the benefits justify that toxicity, Liebmann said. In this case, he said, “for most of the people who voted ‘no,’ the bottom line is that marginal benefit in PFS was not enough to overcome the various downsides.”

While Novartis did not comment directly on the committee’s decision, spokeswoman Dana Cooper said that the company looks forward to the FDA’s final decision by January. — Lena Freund

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.