Briefing documents for Thursday's FDA advisory panel reviewing Rockwell Medical's (RMTI) Triferic iron replacement therapy were posted to the agency's Web site this morning. Overall, the FDA's assessment of Triferic raises questions about the clinical meaningfulness of the data supporting the product's proposed use as an iron replacement and treatment for maintenance of hemoglobin in kidney dialysis patients. This was the big risk for Rockwell Medical going into Thursday's FDA panel, as I described in a column two weeks ago.
Rockwell shares are up 13% to $9.90 Tuesday morning as investors read and digest the FDA's Triferic briefing documents. Why is the stock green if the product is in trouble? Because the FDA review reads fairly straight with little editorializing, which gives the company some hope for a positive vote on Thursday. I have my doubts, but crazier things have happened at FDA advisory panels so we'll see.
FDA and Rockwell agree that the two phase III studies of Triferic both met their respective primary endpoints of maintaining hemoglobin levels compared to placebo with statistical significance. FDA notes, however, that half of the dialysis patients in the phase III studies dropped out before the end of the year-long treatment period. In fact, only 20% of dialysis patients randomized actually finished the full treatment schedule. Most patients who discontinued from the Triferic study did so because they required treatment for anemia with erythropoietin stimulated agents (ESAs) or intravenous iron.
In other words, what's the point of using Triferic if dialysis patients still require ESAs or intravenous iron to maintain hemoglobin levels?
Here's FDA laying out the key efficacy issue for the experts on the panel to discuss Thursday: