Novartis gears up for FDA scrutiny with a new blood cancer drug

Novartis ($NVS) believes its new oncology treatment can make a major difference for patients with multiple myeloma, a blood cancer with a particularly grim prognosis. But first it'll have to convince a group of independent experts that the drug's ability to extend survival by a few months outweighs a significant increase in safety risks.

In documents released ahead of Thursday's meeting of the FDA's Oncologic Drugs Advisory Committee, agency staff spent little time editorializing, focusing instead on the relevant numbers from Novartis' 768-patient Phase III trial. On the efficacy side, a combination of the company's panobinostat, Takeda's Velcade and the steroid dexamethasone prolonged progression-free survival (PFS) by 3.9 months compared to the other drugs on their own, helping patients live a median of one year without a major event. At the same time, 7% of patients in the panobinostat arm died from non-cancer complications compared to just 3.5% in other group, reporting symptoms including myelosuppression, hemorrhage, infection, gastrointestinal toxicity and cardiac toxicity.

And so the task facing FDA advisers is deciding whether the drug's well-demonstrated benefits are worth its serious risks. At the Thursday meeting, the panel will vote on whether to approve panobinostat, which Novartis plans to market as Farydak, handing down an opinion that is commonly--though not obligatorily--followed by the FDA.

Digging into the drug's efficacy data, FDA reviewers point out that PFS results from many patients in the panobinostat were censored due to incomplete and missing assessments. Factoring in all the omitted data, an independent review committee charted a median PFS extension of just 2.2 months over placebo, according to the agency. Separately, while it's too early to make a call on overall survival, the study's secondary endpoint, things are so far trending panobinostat's way, with a median 33.6 months until death in the treatment arm compared to 30.4 months with placebo.

The drug, formerly LBH589, 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. The company has steadily upped the ante on its R&D spend, last year reaching nearly $9.9 billion, 17% of its total revenue.

- read the briefing (PDF)

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