BioMarin Pharmaceutical Inc. (BMRN) A Stock that Could 10x by 2030 on Pipeline Development

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.

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