BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) Price Target Updated: Key Developments in Rare Disease Treatment

• BMO Capital maintained a bullish view on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), with a $97.00 price target that implies significant upside from recent trading levels.
• BioMarin strengthened its leadership by appointing Dr. Robert Plenge, Chief Research Officer and Head of Research at Bristol Myers Squibb, to its Board of Directors.
• The company announced positive Phase 3 clinical trial results for VOXZOGO® in children with hypochondroplasia and submitted a supplemental New Drug Application to the FDA for expanded approval.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is a biotechnology company focused on developing and commercializing therapies for rare genetic diseases. On September 15, 2026, BMO Capital maintained a bullish rating on the company, with a $97.00 price target. Based on a recent stock price near $64.60, that target suggests potential upside of approximately 50.15%.

A price target represents an analyst's estimate of a stock's future value. BMO’s outlook follows its earlier coverage of BioMarin, where the firm highlighted the company’s position as a profitable rare-disease biotechnology business with enzyme therapies, VOXZOGO, and newly acquired Amicus assets. 

BioMarin also strengthened its corporate leadership by appointing Dr. Robert Plenge to its Board of Directors, effective September 10, 2026. Dr. Plenge is Executive Vice President, Chief Research Officer and Head of Research at Bristol Myers Squibb, where he leads scientific work across nine research sites worldwide. His experience in genetics, translational medicine, and drug discovery adds scientific depth to BioMarin’s board as the company expands its rare-disease pipeline. 

The company also reported positive Phase 3 results for VOXZOGO® in children with hypochondroplasia, a rare genetic skeletal condition that affects bone growth. BioMarin said the CANOPY-HCH-3 study met its primary endpoint, with VOXZOGO showing a statistically significant improvement in annualized growth velocity compared with placebo after 52 weeks. The treatment also showed improvements in standing height, height Z-score, and arm span. 

Following the Phase 3 results, BioMarin submitted a supplemental New Drug Application to the U.S. Food and Drug Administration seeking approval of VOXZOGO for children with hypochondroplasia. The company said there are currently no FDA- or EMA-approved medicines for hypochondroplasia, and if approved, VOXZOGO could become the first targeted therapy for this condition, with a potential launch in 2027.