BioMarin's Roctavian US Launch: Focus on Commercial Strategy Amid EU Failures, Analyst Points Out

On Thursday, The FDA approved BioMarin Pharmaceutical Inc's BMRN Roctavian (valoctocogene roxaparvovec-rvox) gene therapy for adults with severe hemophilia A.

Wedbush analyst maintains BioMarin with a Neutral and raised the price target from $69 to $73.

The analyst writes that with the U.S. approval, all focus turns to the U.S. commercial strategy, given the ongoing failed attempt to launch in the EU since receiving EMA approval in August 2022. 

The management intends to offer all payers an outcomes-based warranty that ensures payers would be reimbursed up to 100% of the WAC if the patient does not respond on a "prorated basis" out to 4yr post-dose. 

Management estimates commercial Roctavian would be available in ~2 months, with it taking ~2-5 months for a patient to receive Roctavian after expressing interest and completing eligibility testing potentially.

BMO Capital Markets maintains the Market Perform rating and expect the Roctavian launch to principally drive the BioMarin story in 2023/2024. 

It acknowledges the management saying that treated patients who are satisfied with Roctavian's effect will positively impact Roctavian's further uptake, but this dynamic will be lengthy, and it is currently unclear what portion of patients will be satisfied with Roctavian.

Baird analyst says BioMarin shares traded approximately flat, probably due to some investors shorting the launch and also perhaps due to some minor quibbles regarding the label (weekly ALT monitoring for the first 26 weeks and a ~50% reduction in ALL bleeds on the label, compared to around 80-90%+ reduction in treated, bleeds that BioMarin had generally been focusing in).

It says Roctavian's key upside potential is eventually expanding its market via future label expansions, especially into patients with less severe hemophilia A.

Price Action: BMRN shares are down 1.06% at $87.99 on the last check Friday.