BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the stocks that could 10x by 2030.
Wainwright reiterated a Neutral rating on BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) with a $50 price target. The cautious outlook comes on the heels of BioMarin Pharmaceutical delivering positive Phase 3 Pivotal study results for VOXZOGO. The study met the primary endpoint in children with hypochondroplasia and exceeded expectations, with a highly statistically significant improvement in annualized growth velocity (AGV).
Safety findings were also in line with the established profile in achondroplasia. Hypochondroplasia is a rare genetic disorder characterized by impaired bone growth, resulting in disproportionate short stature and skeletal abnormalities. For the first time, a Phase 3 study of hypochondroplasia has demonstrated impressive gains in growth, including improvements in arm span.