Institute for Clinical and Economic Review (ICER) Base-Case Model Report Results in $4 Million Savings Versus Emicizumab Prophylaxis Per Patient Over a Lifetime

Biologics License Application (BLA) Resubmission to FDA on Track for End of September 2022

SAN RAFAEL, Calif., Sept. 15, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the Institute for Clinical and Economic Review (ICER) released a Draft Evidence Report updating the previous Hemophilia A assessment of the comparative clinical effectiveness and value of ROCTAVIAN ™ (valoctocogene roxaparvovec). 

In the Draft Evidence Report, ICER finds Roctavian is a dominant treatment at an assumed place holder price of $2.5 million, providing substantial cost savings and projected gains in quality adjusted life years. These findings were robust to numerous sensitivity analyses and scenario analyses.  Marking an important milestone in ICER's eight-month process of assessing this treatment, the Draft Evidence Report is now open for public comment, with a final report expected near the end of the year.

In the Draft Evidence Report, ICER calculated the lifetime cost of managing hemophilia A among clinically eligible patients using one-time administration with Roctavian versus emicizumab prophylaxis. Total costs in the model include treatment, treatment-related adverse events, treatment for bleeding episodes, arthropathy, surgery, and non-drug costs.  ICER assumed annual cost of emicizumab to be $640K per year and one-time Roctavian price to be $2.5M. ICER modelled the effect of Roctavian to last 12 years (before patients were assumed to switch back to prophylaxis) for the entire cohort post infusion.  ICER arrived at greater than $4M cost saving per patient over a lifetime with projected improvement in quality of life.   Importantly, ICER incorporated an outcomes-based warranty agreement in its base-case economic model, an innovative approach that BioMarin plans to offer that will allow effective risk sharing for the period of four years and have it ready to implement with payers at launch.  

While Roctavian is approved in the European Union, it is still an investigational therapy in the U.S. and therefore does not have a price. 

"BioMarin is pleased that ICER recognizes the potentially transformative impact of Roctavian as possibly the first gene therapy treatment for severe hemophilia A, and potential to not only deliver profound patient benefit, but also potential long-term healthcare savings," said Jeff Ajer, Executive Vice President, Chief Commercial Officer at BioMarin. 

BioMarin remains committed to bringing Roctavian to eligible patients with severe hemophilia A in the United States and is targeting a Biologics License Application (BLA) resubmission for Roctavian by the end of September 2022. Typically, BLA resubmissions are followed by a six-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.

BioMarin expects first commercial sales in Europe in the fourth quarter of 2022.

ICER is an independent non-profit research organization that evaluates the evidence on the effectiveness and economic value of prescription drugs and other medical devices. The Draft Evidence Report will next be evaluated by one of ICER's three independent evidence appraisal committees before the organization issues its final Evidence Report expected near the end of the year.

Safety Summary

Overall, single 6e13 vg/kg dose of Roctavian has been well tolerated with no delayed-onset treatment related adverse events. The most common adverse events (AE) associated with Roctavian occurred early and included transient infusion associated reactions and mild to moderate rise in liver enzymes with no long-lasting clinical sequelae. Alanine aminotransferase (ALT) elevation (113 participants, 80%), a laboratory test of liver function, remained the most common adverse drug reaction. Other adverse reactions included aspartate aminotransferase (AST) elevation (95 participants, 67%), nausea (52 participants, 37%), headache (50 participants, 35%), and fatigue (42 participants, 30%). No participants developed inhibitors to Factor VIII, thromboembolic events or malignancy associated with Roctavian.

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (Factor VIII levels