- BioMarin has 6 commercialised assets in the rare disease space, targeting e.g. lysosomal storage diseases.
- The company is on track for ~$1.8bn of revenues in FY21, and importantly, a small non-GAAP net income, reversing years of heavy losses.
- The company also has a PDUFA date on November 30th when the FDA will decide whether to approve its candidate Voxzogo for Achondroplasia.
- The trial data - and an approval in Europe - suggests Voxzogo will get over the line, and the therapy has blockbuster potential.
- Another major catalyst is hemophilia therapy Roxavian. Rejected by the FDA last year, BioMarin may now have the data it needs for an approval, and multi-billion market in the US and EU. I am bullish.
For further details see:
BioMarin: Voxzogo Approval Could Trigger A Lengthy Bull Run For Cash Rich Biotech