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BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the stocks that could 10x by 2030. On May 22, H.C. 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. Consequently, BioMarin Pharmaceutical intends to submit a supplemental New Drug Application to the US Food and Drug Administration in the third quarter. It also plans to make submissions to the European Medicines Agency.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is a global biotechnology company that develops and commercializes innovative therapies for rare and ultra-rare genetic diseases. It is a pioneer in enzyme replacement therapies (ERT) and gene therapy.

While we acknowledge the potential of BMRN as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.

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