Rockwell Dialysis Therapy Wins Panel Support but Final FDA Approval? Not So Fast.

BOSTON ( TheStreet) -- An FDA advisory panel Thursday evening decided the benefits of Rockwell Medical's (RMTI) iron replacement therapy Triferic outweighed its risks for maintaining iron stores and hemoglobin in kidney dialysis patients. The panel vote was 8-3 in favor of recommending Triferic's approval. As you'd expect, Rockwell Medical shares are up 25% to $11.50 in pre-market trading Friday. 

Going into Thursday's panel, I believed the FDA's invited experts would vote against Triferic for two big reasons. First, the design of the clinical trials supporting the product were deeply flawed and didn't reflect the way dialysis patients are treated in the real world. Second, the benefit demonstrated by Triferic in terms of managing hemoglobin and anemia were marginal and I would argue, clinically meaningless.

But I was wrong. The outcome of the Triferic FDA panel was far different from what I expected. Being wrong isn't pretty. Being wrong about Rockwell and Triferic is particularly frustrating because much of Thursday's proceeding were devoted to the exact concerns I've cited. Certain panel members raised repeated objections to the design of the Rockwell clinical trials, which compared Triferic against a placebo (highly unusual) and didn't allow dialysis patients to use intravenous or oral iron to manage their anemia (which goes against standard medical practice.)

During one exchange, a panel expert said, "The way you [Rockwell] are proposing we use this [Triferic] is not the way you used it in the clinical trial." 

Earlier, another panel member asked, "How would I dose [Triferic] in the real clinical setting? There is no guidance based on the clinical data."

The FDA's medical reviewers called the Rockwell clinical trial design "contrived" and complained that results from the study were clouded because only 20% of the enrolled patients made it through the mandated 48 weeks of treatment.

Rockwell even admitted that its clinical trials were entirely dis-engaged from the way doctors and nurses managed hemoglobin and anemia in dialysis patients. The company insisted that it was impossible to run a clinical trial of Triferic in a "real world setting." Several panel members disagreed, and so did the FDA. 

And then, the panel members voted 8-3 to recommend Triferic's approval. 

Pardon my language, but W...T...F....?

So, I was wrong about the Triferic FDA panel. I predicted a negative vote. It was positive. Rockwell Medical's stock price will be higher Friday. 

The story is not over. FDA did an awful job conveying its concerns about the Triferic data to the panel's experts. Triferic was reviewed by the FDA's Oncologic Drugs Advisory Committee (ODAC), supposedly one of the most intelligent and toughest review divisions inside the FDA. ODAC is the dominion of Dr. Richard Pazdur, the FDA executive biotech and drug companies fear most. Yet Pazdur barely managed to speak during the Rockwell session of the ODAC panel, and his assembled team of reviewers were, shall I say, a tad inept.

With the panel over, the next step will be FDA's final approval decision, expected on Jan. 24. FDA panels are advisory only so the agency is not required to go along with the recommendations. FDA has and does reject drugs which received positive panel votes. 

On Jan. 24, if logic means anything, FDA should reject Triferic with a demand that the company conduct a proper clinical trial which reflects the real way iron replacement therapy is used in dialysis patients. I'm hopeful the FDA will eventually reject Triferic. To put it mildly, Thursday's panel decision was wrong. 


Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.

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