BioMarin (BMRN) has received European approval for its gene therapy for Hemophilia A, explains John McCamant, biotech sector expert and editor of The Medical Technology Stock Letter.

The European Commission (EC) granted conditional marketing authorization (CMA) to Roctavian (6e13 vg/kg), the first gene therapy approved for adults with severe hemophilia A.

The label is specific for Hem A patients without factor VIII inhibitors or detectable antibodies to AAV5 (with a FVIII/ALT monitoring requirement and thrombosis/vector integration advisory), and 10-year market exclusivity per the orphan drug designation.

BMRN proposed a net price of $1.5 million and initial sales are expected to start in Q4:22. The launch should be interesting as BMRN estimates 3,200 eligible patients within its EMEA footprint, with Germany and France as key initial markets.

In our view, Roctavian’s clinical profile suggests long-term durability of benefit on bleeding rates and benign safety. This long-term durability will be key as BMRN is utilizing an outcomes-based payment approach to address potential payor concerns regarding initial patient response and durability.

For example, durability of benefit is determined by patient’s not needing to return for prophylaxis during the guarantee period; for example, in the case of a five-year guarantee period, the resumption of prophylaxis FVIII following year 4 would imply 80% value capture, with BMRN to rebate back 20%.

In the US, we remain optimistic on approval post the BLA resubmission in September with a decision expected in H1:23. 

Of interest, on the conference call BMRN indicated that Roctavian’s eventual US price will be $2-2.5 million which illustrates two important points; a) that biotech drugs will retain premium pricing despite recent Medicare reforms and; b) that the U.S. market will retain it’s higher drug prices when compared to Europe.

BioMarin is on a roll with three new products being or about to be launched. BMRN is a buy under $100 with a target price of $150 per share.

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