BioMarin Pharmaceutical Inc. (BMRN)

[Fezziwig2008]

$BAC On $BMRN

“FDA has requested BioMarin provide 2-year annualized bleed rate (ABR) data for Roctavian to complete the filing package (which BioMarin expects to have in November 2021). 6-month phase 3 data showed 85%/100% reduction in mean/median ABR and 4- year phase 1/2 data showed a 95%/100% reduction, which gives us comfort in the reproducibility in 2-year phase 3 data. Notably, in the phase 1/2 dataset, the 2-year mean bleed rate improvement was better than in year 1 (99% vs. 92%) so we could see the 85% reduction potentially improve. If Roctavian can show anything near this in November 2021, we think BioMarin should have no problem getting approval.”