Panobinostat could be the next potential myeloma therapy approved by the U.S. Food and Drug Administration (FDA).

Moreover, an approval decision related to the drug's potential marketing in the United States could be made by this fall.

In a surprisingly indirect manner, Novartis, the Swiss pharmaceutical com­pany that is developing pan­o­bino­stat ^[1] (LBH589), announced this morn­ing that it has filed an application to have pan­o­bino­stat approved as a new drug by the FDA.

The announcement was indirect in the sense that Novartis did not actually say it has filed an application for FDA approval of pan­o­bino­stat.

Instead, the company said that the FDA last month granted pan­o­bino­stat "priority review."

The FDA, however, only grants a drug priority review – instead of standard review – once an application has been submitted for the drug to be reviewed and potentially approved by the FDA. A Novartis spokesperson also confirmed to The Beacon that pan­o­bino­stat is currently under review by the FDA, and that the new drug application was filed in March.

Thus, the key news in the Novartis announcement – from the perspective of the multiple myeloma com­mu­nity – is that the company has filed the drug with the FDA for review and potential approval.

Of course, the news that pan­o­bino­stat has been granted priority review is also an important de­vel­op­ment. The target FDA review time for applications granted a priority review is six months, or four months shorter than for drugs with a standard review designation.

Given that pan­o­bino­stat new drug application was filed in March, an FDA decision on the application can be expected by September (six months from March).

Although the Novartis announcement this morning did not provide many details about its application for FDA approval of pan­o­bino­stat, a Novartis spokesperson confirmed to The Beacon that the application is for approval as a new myeloma treat­ment.  In addition, the application is based on the data for pan­o­bino­stat presented at this morning's session of myeloma-related pre­senta­tions at the 2014 American Society of Clinical Oncology (ASCO) meeting.

Those data are from the Phase 3 clinical trial known as PANORAMA-1. The trial tested pan­o­bino­stat in com­bi­na­tion with Velcade ^[2] (bortezomib) and dexamethasone ^[3] (Decadron) in relapsed/refractory multiple myeloma patients who have failed at least one prior treatment.  The presentation of the PANORAMA-1 results was discussed in the Beacon's ASCO 2014 myeloma preview ^[4], and further details will be provided in a future Beacon ASCO update.

The Novartis announcement also did not provide any specific updates on filings for the approval of pan­o­bino­stat in other parts of the world, saying only that "additional global regulatory submissions are underway" -- which, it should be noted, could mean that some filings outside the U.S. already have been made.

Estimates provided to The Beacon by Evaluate Ltd., a market intelligence and analysis firm, suggest that an application for the drug's approval in Europe could be filed some­time this year.  This would result in an approval decision in Europe by the end of next year (2015).

Panobinostat is an oral drug that belongs to a class of drugs called histone deacetylase (HDAC) inhibitors, which work by increasing the production of proteins that slow cell division and cause cell death.

Preclinical studies have shown that pan­o­bino­stat in combination with Velcade more effectively kills myeloma cells than either drug alone.

Another HDAC inhibitor, Zolinza ^[5] (vorinostat), has also been studied as a potential new treatment for multiple myeloma.  However, it was recently shown to have had only a small impact on progression-free survival when used in combination with Velcade in relapsed/refractory myeloma patients (see related Beacon ^[6] news).

For more information about the pan­o­bino­stat trial results presented this morning and pan­o­bino­stat's regu­la­tory status, see the Novartis press release ^[7].