Experimental Hemophilia Therapy May Cost Between $2 and $3 Million

An experimental gene therapy for patients with hemophilia A may be priced between $2 and $3 million, according to The Wall Street Journal. Valoctocogene roxaparvovec (Valrox, BioMarin Pharmaceutical Inc) would be the first gene therapy in the United States for the most common form of hemophilia if approved by the FDA.¹

Two gene therapies are currently approved in the United States, including onasemnogene abeparvovec (Zolgensma, Novartis), which is the most expensive drug in the world at $2.1 million. The new therapy from BioMarin may replace onasemnogene abeparvovec in that position.¹

According to The Wall Street Journal, BioMarin Chief Executive Jean-Jacques Bienaime said the eventual list price of valoctocogene roxaparvovec should be compared with annual costs of existing therapies and the lifetime cost of treatment, which the company estimated to be at $25 million.¹

Although BioMarin Pharmaceuticals has not announced a price for the therapy, according to Bienaime they have spoken to insurer, who indicated that they are comfortable with the $2 to $3 million range. Bienaime said the company is open to alternative payment structures for the drug, including pay-for-performance, according to The Wall Street Journal.¹

Another treatment for patient with hemophilia, emicizumab-kxwh injection (Hemlibra, Genentech Inc), was approved by the FDA in 2018.² The annual list price for the drug is approximately $492,000 per year for a patient of average weight with hemophilia A. The initial first-year dose costs an additional $38,000.³

BioMarin submitted its biologic license application to the FDA in December 2019,⁴ and does not expect to hear any results until at least late August of 2020 according to The Wall Street Journal.¹

REFERENCES

• Hopkins J. BioMarin Explores Pricing Experimental Gene Therapy at $2 Million to $3 Million. Wall Street Journal. https://www.wsj.com/articles/biomarin-explores-pricing-experimental-gene-therapy-at-2-million-to-3-million-11579190318?ns=prod/accounts-wsj/. Accessed Jan 20, 2020.
• FDA Approves Emicizumab-kxwh for Hemophilia A with or without factor VIII inhibitors [news release]. Washington DC; Dec 14, 2018. FDA website. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-emicizumab-kxwh-hemophilia-or-without-factor-viii-inhibitors. Accessed Jan 20, 2020.
• Miller J. US FDA expands approval of Roche hemophilia drug. Reuters. https://www.reuters.com/article/us-roche-hldg-hemlibra-approval/us-fda-expands-approval-of-roche-hemophilia-drug-idUSKCN1ME29T. Accessed Jan 20, 2020.
• BioMarin Submits Biologics License Application to US Food and Drug Administration for Valoctocogene Roxaparvovec to Treat Hemophilia A [news release]. San Rafael, CA; Dec 23, 2019. BioMarin website. https://investors.biomarin.com/2019-12-23-BioMarin-Submits-Biologics-License-Application-to-U-S-Food-and-Drug-Administration-for-Valoctocogene-Roxaparvovec-to-Treat-Hemophilia-A/. Accessed Jan 20, 2020.