FDA Says Yes to Rockwell Medical's Iron Replacement Drug

Published: Jan 27, 2015

FDA Says Yes to Rockwell Medical's Iron Replacement Drug
January 26, 2015
By Jessica Wilson, BioSpace.com Breaking News Staff

Rockwell Medical announced today that the U.S. Food & Drug Administration (FDA) has approved its drug Triferic for commercial sale as an iron replacement. The drug is intended for use by adult patients with hemodialysis dependent chronic kidney disease in order to maintain their hemoglobin.

“We see Triferic as a paradigm shift in the treatment of anemia,” Raymond Pratt, Chief Medical Officer of Rockwell, said in a statement. “Importantly, Triferic is the first product that can safely allow dialysis patients to maintain target hemoglobin without the need for IV iron. Data suggests that we have been overloading our dialysis patients with IV iron, and this is an increasing concern to the hemodialysis community. Triferic offers a more physiologic way to deliver and maintain iron balance in hemodialysis patients.”

The clinical trial upon which the FDA based its approval included more than 1,400 patients who were treated with Triferic. The results showed Triferic to be an “an effective and highly-differentiated iron delivery therapy with a safety profile similar to placebo,” according to the company.

During dialysis, patients tend to lose 5-7 milligrams (mg) of iron per session, which Triferic is intended to replace, according to Reuters. It is the first drug to allow dialysis patients to be treated without the need for intravenous iron. The drug works like dietary iron by delivering iron directly to the bone marrow, Stifel Nicolaus analyst Annabel Samimy told Reuters. “Iron administered intravenously is less efficient as it is encased in a shell, which goes into the liver before being delivered and gets blocked by the immune system,” she said, and estimated that Triferic would hit sales of $200 million-$250 million in the United States.

When the FDA advisory panel voted in November 2014 to recommend Triferica’s approval, an analyst at The Street, Adam Feuerstein expressed surprise and concern about the decision. “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,” Feuerstein wrote, citing his extended analysis of the data in an earlier article. “The benefit demonstrated by Triferic in terms of managing hemoglobin and anemia were marginal and I would argue, clinically meaningless.”

His analysis was based on concerns raised by the FDA in briefing documents released before the advisory meeting in November 2014 that stated, “There is a considerable amount of ‘missing data’ due to study drug discontinuation by the end of the study treatment period.”

“It is noted that a large portion of the missing data due to withdrawn patients is due to protocol-mandated changes in anemia management, which were incorporated into the protocol to protect patient safety,” said the FDA. “The large proportion of patients with early withdrawal from study treatment necessitates examination of the study data to assess impact of the early discontinuation of patients on the validity and robustness of the efficacy results.

Despite this, the advisory committee voted to recommend the drug 8-3, and while the FDA is not required to follow the committee’s recommendation, it often does.

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