Baxter International Inc. has scored a win from the U.S. FDA, which has granted the de novo application for Theranova, the company’s novel dialysis membrane.

For his part, Cowen’s Josh Jennings noted that de novo designations are rare in the dialysis space, echoing a company statement. “We think the de novo classification strengthens any argument for the uniqueness of the technology, which should help its commercial experience,” he added. “BAX had expected an FDA clearance in [second half of] '20, so this action falls on the early end of that range.”

What differentiates the offering from Deerfield, Ill.-based Baxter? It was designed to deliver expanded hemodialysis (HDx) therapy, which filters a wider range of molecules from the blood than traditional hemodialysis filters. It targets the removal of conventional and large middle molecules. These middle molecules may be associated with inflammation and cardiovascular disease in patients with kidney failure.

HDx is done the same way as conventional HD, with the only requirement being a change of the dialyzer membrane. The dialyzer’s medium cut-off membrane combines high permeability and selectivity for uremic toxins, retaining essential proteins and maintaining albumin levels during treatment. This profile helps expand clearance, allowing for filtration closer to that of the natural kidney, the company said, citing published data.

“Individually, the side effects from standard HD, which patients typically undertake three days a week, four hours per day, may seem manageable. However, the chronic effects of treatment accumulate and over time, cause some patients to give up on therapy,” explained Mary Gellens, nephrologist and senior medical director at Baxter. “HDx therapy enabled by Theranova is a promising alternative to what is currently available because it delivers a filtration profile that is closer to the natural kidney.”

Gavin Campbell, general manager of Baxter’s U.S. Renal Care business, told BioWorld that the product is available for order now, and clinics nationwide will see delivery in about a week. While he noted that the company does not comment on specific product pricing, “our goal is to make Theranova accessible for all HD patients in the U.S., because Theranova is unlike any other dialyzer, as it delivers HDx therapy,” he added.

He further explained that the clinics across the globe have adopted Theranova, with more than 3 million treatments completed as of May. “Theranova is used in more than 850 clinics, across 43 countries,” he confirmed.

Data backing the product

To obtain the FDA’s nod, Baxter conducted a randomized controlled clinical study in the U.S. that evaluated the safety and efficacy of HDx therapy enabled by Theranova. Results were discussed during the 2019 American Society of Nephrology Kidney Week, with 172 hemodialysis patients receiving therapy with either the Theranova 400 or a high-flux dialyzer ELISIO-17H over 24 weeks of treatment, with a primary efficacy endpoint measuring the reduction ratio of lambda (λ) free light chains at 24 weeks of treatment, while maintaining predialysis serum albumin levels.

Data from the study, which was published in the August 2020 Clinical Journal of the American Society of Nephrology, found that expanded hemodialysis therapy with the Theranova 400 dialyzer gave superior removal of large middle molecules, as exemplified by λ free light chains, compared to a similarly sized high flux dialyzer while maintaining serum albumin levels. Currently available dialysis technologies offer limited clearance of these molecules, the company noted.

Reimbursement questions

In early July and ahead of the FDA’s decision, CMS issued the proposed rule titled “CY 2021 Changes to the End-Stage Renal Disease (ESRD) Prospective Payment System and Quality Incentive Program.” CMS noted that Baxter had requested a transitional add-on payment adjustment for new and innovative equipment and supplies for Theranova.

CMS wrote that the technology was promising, but there was insufficient evidence at the time “to demonstrate a clear clinical benefit for Medicare dialysis patients.” With that said, the agency said that more evidence might come during the proposed rule’s comment period. “We expect the final rule to be issued later this year,” Wells Fargo’s Larry Biegelsen wrote at the time in a note that said the add-on payment appeared uncertain. “An add-on payment for Theranova represents upside to our estimates. Theranova is to be booked in BAX’s US hemodialysis (HD) business which we estimate will generate revenues of $204M (- 2%) in 2020.”

Subsequently, during the company’s July 30 second-quarter earnings call, David Lewis with Morgan Stanley asked about reimbursement expectations for Theranova. CEO Joe Almeida said the company would submit comments to the CMS proposed rule by Sept. 4, addressing issues raised by the agency. “I can't speak on behalf of CMS. My confidence is very high that we'll be able to address the questions that they posed as well with meta-analysis that we're doing, but that doesn't mean that they're going to make a decision one way or the other, I can't predict that,” he added.

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