BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) is one of the best healthcare stocks with the highest upside. In a report released on October 14, Cantor Fitzgerald analyst Olivia Brayer reiterated a Buy rating for BioMarin Pharmaceutical Inc. (NASDAQ:BMRN). However, the analyst slashed the firm’s price target on the stock from $90 to $80. BioMarin Pharmaceutical Inc. (BMRN): Among Small-Cap Healthcare Stocks Hedge Funds is Buying J.P. Morgan analyst Jessica Fye also maintained a Buy rating on BioMarin Pharmac ...

Core Insights - New data indicates that treatment with VOXZOGO significantly improves tibial bowing in children with achondroplasia, addressing a common source of pain and functional impairment [1][2] - A large observational study revealed higher rates of comorbidities, surgeries, and doctor visits in individuals with hypochondroplasia compared to those without [4] Group 1: VOXZOGO Treatment Efficacy - VOXZOGO treatment resulted in a significant reduction in tibial bowing in children compared to placebo, with sustained improvements observed over several years [2] - Continuous and early treatment with VOXZOGO is associated with sustained increases in annualized growth velocity (AGV), potentially leading to a height increase of 21.7 cm in girls and 26.4 cm in boys when treated from 6 months until final adult height [3] Group 2: Hypochondroplasia Insights - Research on hypochondroplasia indicates that individuals experience significantly higher rates of comorbidities and increased healthcare utilization, including more frequent doctor visits and surgeries [4][5] - Early diagnosis of hypochondroplasia is emphasized as critical for timely intervention and management [5] Group 3: Ongoing Research and Development - BioMarin is advancing its CANOPY clinical program for VOXZOGO, with ongoing studies in various conditions including hypochondroplasia and Turner syndrome, and plans to share topline data in 2026 [7] - VOXZOGO is currently approved in multiple regions for increasing linear growth in children with achondroplasia, with ongoing evaluations to confirm clinical benefits [15][19]