NICE blames BioMarin for Brineura rejection after price talks break down

BioMarin was “unable to address concerns about long-term effectiveness" of Brineura, NICE says. (BioMarin)

BioMarin and England’s cost-effectiveness gatekeeper have been negotiating for a year, but that hasn’t helped the rare-disease specialist secure reimbursement for its Batten disease drug Brineura.


Friday, the National Institute for Health and Care Excellence (NICE) published draft guidance sticking with its earlier decision not to endorse Brineura in children with the disease, an inherited nervous system disorder that affects between one and six babies each year in the U.K.


The reason? According to NICE, BioMarin was “unable to address concerns about long-term effectiveness” of the product, and after a year of haggling, “the company was unable to price the treatment at a level that would have addressed the problems highlighted during NICE’s assessment of it.”


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The watchdog turned down Brineura even after recognizing that Brineura is “an important development for treating” Batten disease that’s “shown substantial short-term benefits in slowing the rate at which it progresses.”


And in doing so, it placed the blame squarely with BioMarin. “We and NHS England have been very clear with the company about what would be needed in order for us to be able to recommend,” Brineura, Meindert Boysen, director of NICE’s Centre for Health Technology Evaluation, said in a statement, noting that the body gave the drugmaker “ample opportunity to come up with a workable solution.”

BioMarin’s window to serve up a NICE-approved discount hasn’t completely closed, however. The company, healthcare professionals and others now have the chance to consult on the guidance and appeal the draft recommendations.

Meanwhile, industry watchers seem more focused on BioMarin’s pipeline prospects than its reimbursement woes. “Investors/analysts remain obsessed over whether we’ll see waning” factor VIII activity levels with hemophilia A candidate valoctocogene roxaparvovec after three years of follow up, Evercore ISI analyst Josh Schimmer wrote in a recent note to clients.

“BMRN has a strong and growing base business and the rest of the pipeline continues to advance rapidly,” he added.