ICER says it’s fair for BioMarin and CSL to price incoming hemophilia gene therapies at $2M-$3M

Get ready for million-plus-dollar gene therapies to become the norm rather than the exception, and in some cases, the Boston-based drug pricing watchdog ICER is coming on board.

The nonprofit ICER (Institute for Clinical and Economic Review) on Wednesday unveiled a new report finding that CSL Behring’s potential gene therapy etranacogene dezaparvovec for hemophilia B, which is due for an FDA approval decision by the end of this month, could be fairly priced at around $3 million. ICER also updated its previous assessment of BioMarin’s EU-authorized hemophilia A gene therapy Roctavian, which ICER said could be fairly priced at about $2 million.

David Rind

“The new gene therapies can result in successfully-treated patients appearing ‘cured’ for at least a period of time,” ICER chief medical officer David Rind said in a statement. “During this period these gene therapies will eliminate the need for expensive prophylactic treatment. However, the duration of this ‘cure’ and the safety of therapies remain important uncertainties.”

In the case of CSL’s hemophilia B candidate, which uses an AAV5 vector to carry the Padua gene variant of Factor IX (FIX-Padua), generating FIX proteins that are 5x-8x more active than normal, ICER noted that treated patients saw an 80% reduction in treated joint bleeds and similar reductions in other bleeds when compared with their bleeding rates prior to gene therapy.

While no patients successfully treated with the gene therapy returned to prior prophylactic treatment during the first 18 months on the hemophilia B therapy, ICER said it “is not yet clear that the initial increase in factor IX levels will be maintained for decades, though the results are encouraging. Finally, the reduction in burden of therapy – no longer needing weekly or more frequent IX factor therapy is a major benefit for patients.”

About 30,000 Americans have either hemophilia A or B, which means they have an increased tendency to bleed due to inherited deficiencies of factor VIII and factor IX.

In the case of BioMarin’s Roctavian, or valrox, which has already been through a round of rejection at FDA and faces a new PDUFA date of March 31, 2023, ICER concluded that there is “low certainty” about the net health benefit for Roctavian compared with Roche’s Hemlibra (emicizumab), which costs about $630,000 per year.

In the EU, however, Roctavian has already won a conditional approval and set its price at about $1.5 million.

But ICER says BioMarin could go even higher with that price, to almost $2 million. And at a cost of $2.5 million, if the durability of the therapy holds up for four years, ICER notes that valrox goes from not cost-effective to dominant.

“The gene therapies have large cost savings associated with them with very large lifetime costs associated with both the treatments and comparators in both models,” ICER concluded. “In addition, the gene therapies are associated with slightly higher QALYs and lower bleeds.”

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