Cost watchdog ICER backs multimillion-dollar price tags for BioMarin, CSL hemophilia gene therapies

The high cost of gene therapies has proved a sticking point for the personalized medicines in recent years, most notably prompting bluebird bio to withdraw its blood disorder treatment Zynteglo in Europe after running into an ‘untenable’ pricing situation with the bloc.

But bluebird’s EU price for the one-time beta-thalassemia therapy—originally launched at $1.8 million in June 2019—could become the new norm in the U.S. And that’s on the low end.

Wednesday, Boston’s Institute for Clinical and Economic Review (ICER) rolled out an evidence report focusing on the clinical effectiveness and value of CSL Behring’s etranacogene dezaparvovec for hemophilia B. At the same time, the U.S. drug cost watchdog updated its previous hemophilia A assessment on BioMarin’s valoctocogene roxaparvovec, which recently clinched approval in Europe as Roctavian.

ICER’s assessment found CSL’s med—up for an FDA priority review decision at the end of November—would be fairly priced at upwards of $2.9 million. BioMarin’s Roctavian, which the company resubmitted to the U.S. FDA in late September, would be acceptable at a cost of approximately $1.9 million.

Still, “[d]uration of benefit with gene therapies and risks for rare side effects remain major uncertainties,” ICER cautioned in a release.

The watchdog’s review comes shortly before a virtual public meeting on Nov. 18, when an ICER committee will review the evidence on the meds and hear further testimony. The experts will also deliberate over the treatments’ comparative effectiveness, plus other potential benefits and long-term value.

But in its own review, ICER was largely onboard with high million-dollar price tags for both BioMarin's and CSL's meds. Both therapies show “health gains and the potential for substantial cost offsets due to elimination of the need for very expensive prophylactic treatment."

As for the greater gene therapy pricing landscape, Roctavian’s net price in Europe stands at around 1.5 million euros (about $1.46 million), BioMarin said this summer. While the med would still be reasonably priced at up to $1.9 million, according to ICER’s latest report, BioMarin could go as high as $2.5 million.

BioMarin has said it expects to reap its first commercial sales from Roctavian in Europe during the fourth quarter.

As for another gene therapy player, bluebird is getting a second bite at the gene therapy apple after the U.S. FDA approved its $2.8 million treatment Zynteglo in August. Bluebird believes that hefty price tag “reflects the value of a one-time treatment in the U.S.,” the company’s chief commercial officer, Tom Klima, told Fierce Pharma earlier this year.

And right on the heels of Zynteglo’s U.S. green light, bluebird in September clinched an approval for eli-cel—now christened Skysona—in the rare neurological disorder cerebral adrenoleukodystrophy (CALD). The company is charging $3 million per treatment with Skysona, higher than Zynteglo’s $2.8 million.

While gene therapies are no doubt changing the treatment landscape for various cancers and rare diseases, the potential for “cost shocks” abound, Brett Sahli, assistant vice president of pharmaceutical trade relations at Prime Therapeutics, said on a recent Fierce panel.

The challenge is even greater globally, ICER president Steven Pearson, M.D., M.Sc., added on the panel, noting gene therapy is still a “fairly dicey business proposition” for companies in the field. Pricing for therapies in the millions of dollars makes access “impossible” in many places, Pearson said, “unless it's on a completely compassionate or other basis outside the developed countries.”