Rockwell clears an FDA hurdle with its dialysis drug

A group of FDA advisers voted in favor of approving Rockwell Medical's ($RMTI) iron-replacement treatment for dialysis patients, setting aside concerns about the drug's pivotal data and burnishing the company's hopes of finally launching its top prospect.

The agency's Oncologic Drugs Advisory Committee voted 8-3 that Triferic, a drug designed to noninvasively deliver iron to the bone marrow of dialysis patients, has benefits that outweigh its risks, recommending full approval. Rockwell's treatment met its primary endpoints of boosting hemoglobin and serum iron in chronic kidney disease sufferers across two Phase III trials, and, despite some repeatedly noted safety concerns, that was enough to win over the majority of the committee.

The final decision now falls to FDA staff, which is not beholden to follow the panel's vote but commonly does so. The agency is expected to weigh in on Triferic by Jan. 24.

Rockwell CEO Robert Chioini

"We welcome the committee's recommendation supporting a positive benefit/risk of Triferic," Rockwell CEO Robert Chioini said in a statement. "We look forward to working with the FDA to complete its review of Triferic. We remain committed to addressing the unmet needs in managing patients with chronic kidney disease."

The panel's recommendation contrasts with the markedly dimmer view of Triferic taken by FDA reviewers. In documents posted ahead of Thursday's meeting, agency staff acknowledged the drug's success in meeting its efficacy endpoints but also flagged an alarmingly high drop-out rate and questioned whether Rockwell's analysis adequately demonstrated the drug's effect. Fewer than 20% of patients made it all the way to either study's 48-week end mark, and about half didn't even get to 20 weeks. That led to "a considerable amount" of missing data, reviewers wrote, casting doubts on "the validity and robustness of the efficacy results."

Furthermore, Rockwell handed in Phase II data designed to demonstrate that Triferic reduced the need for erythropoiesis-stimulating agents (ESAs), drugs designed to spur the production of red blood cells, but agency staff was unconvinced by the results, pointing out that while Triferic contributed to a reduction in prescribed doses of ESA, actual dosing wasn't significantly different.

Those issues, discussed but ultimately overlooked by the panel, could still stand in the way of Triferic's eventual approval, let alone commercial adoption.

Rockwell's shares jumped more than 20% overnight following the vote.

- read the statement