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Hemophilia gene therapies from BioMarin, CSL pick up an early ICER endorsement

Even at a hefty price tag of $2.5 million per treatment, oncoming gene therapies can be the most cost-effective way for patients with hemophilia types A and B to deal with their condition.

That’s the conclusion of the Institute for Clinical and Economic Review (ICER) in a draft evidence report released on Tuesday.

Over the last four months, the independent organization has compared BioMarin’s Roctavian for hemophilia A and CSL Behring’s etranocogene dezparovec for hemophilia B against commonly used therapies for the bleeding disorders.

Roctavian and etranocogene dezparovec are not only more effective, ICER said, but in the long run they’re also less expensive.

“The gene therapies have large cost savings associated with them, with very large lifetime costs associated with both the treatments and the comparators,” ICER wrote. “In addition, the gene therapies are associated with higher (quality of life scores) and lower bleeds.”

Neither of the gene therapies have been approved by the FDA. But last month, European Union regulators blessed Roctavian for those with a severe form of type A, which is the more common version of hemophilia.

Meanwhile, in May of this year, the FDA designated etranocogene dezparovec for priority review. The gene therapy was developed by uniQure and is being commercialized by CSL Behring.

In Europe—where regulators thoroughly scrutinize price before granting marketing authorization—BioMarin will charge approximately $1.5 million for a dose of Roctavian, but it is likely to be priced higher in the United States. As for etranocogene dezparovec, CSL has yet to disclose a price.

In its analysis, ICER assigned a “placeholder” price of $2.5 million for each of the one-time treatments, based on the average price of other gene therapies. ICER compared this figure to other drugs currently used combat hemophilia such as CSL LImited’s Idelvion, which goes for $753,353 annually, Bioverativ’s Alprolix ($744,303), Novo Nordisk’s Rebinyn ($713,552), Genentech’s Hemlibra ($639,543) and Pfizer’s Benefix ($565,391).

Based in part on the lifetime cost of comparison drugs, which can reach into the millions of dollars, ICER endorsed the gene therapy prospects.

As for efficacy, ICER studied trial results for both gene therapies that showed their benefits in controlling bleeding.

“The primary benefit from gene therapy is a reduction in annualized bleeding rate (ABR) over time,” ICER wrote about etranocogene dezparovec, which was investigated in CSL Behring’s Hope-B trial. “Bleeding into joints is particularly important as repeated bleeding events lead to progressive joint damage and thus progressive disability and pain.”

The trial showed reductions of 80% in treated joint bleeds and 64% in bleeds overall, when etranocogene dezparovec was compared to CSL Limited’s factor IX inhibitor Idelvion.

“A secondary benefit of gene therapy is freedom from the need of injecting factor IX into a vein one or more times a week,” ICER wrote.

Trial results comparing Roctavian with other hemophilia A drugs revealed similar advantages in the reduction of bleeding.

But ICER also warned on the limitations of trials that had occurred over such a relatively short period of time—especially when assessing the value of the treatments over a projected lifetime.