10 Most Promising Stocks with Highest Upside Potential

7. BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)

Average Upside Potential: 55.42%

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the most promising stocks with highest upside potential. On June 16, BioMarin presented new clinical data at ENDO 2026, highlighting progress in its treatments for skeletal dysplasias. An investigator-led Phase 2 extension study showed that three years of treatment with VOXZOGO (vosoritide) resulted in sustained improvements in annualized growth velocity and height standard deviation scores for children with hypochondroplasia.

These findings build on recent positive Phase 3 results, with the company planning to submit a supplemental NDA to the FDA in Q3 2026. The company also shared Phase 1 data for BMN 333, an investigational long-acting C-type natriuretic peptide/CNP for achondroplasia. Results from a single-ascending dose study showed that BMN 333 provided sustained systemic exposure, supporting a potential weekly dosing schedule.

The treatment was well tolerated, and its ability to significantly increase free CNP exposure compared to existing therapies suggests it could become a new standard of care. Enrollment is currently underway for a registration-enabling Phase 2/3 study. These developments reinforce BioMarin Pharmaceutical Inc.’s (NASDAQ:BMRN) commitment to addressing growth-related skeletal conditions where few or no approved medical treatments currently exist.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) develops and commercializes therapies for serious and life-threatening medical conditions and rare diseases. The company’s product pipeline includes Valoctocogene roxaparvovec, Vosoritide, and BMN 307.