BioMarin: Ph3 win: Voxzogo clears Ph3 hypochondroplasia trial with robust effect

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BioMarin: Ph3 win: Voxzogo clears Ph3 hypochondroplasia trial with robust effect

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BioMarin

Ph3 win: Voxzogo clears Ph3

hypochondroplasia trial with robust effect

Maintain Rating: BUY | PO: 80.00 USD | Price: 50.20 USD

HCH could add $250m+ sales even with pipe competitors 20 May 2026

This evening, BMRN announced positive Ph3 topline of Voxzogo in hypochondroplasia Equity

(HCP). The study met the primary endpoint on annualized growth velocity (AGV), where

Jason M. Gerberry

effect size of 2.33cm/year compared to placebo and above ~1.5cm/year range we heard Research Analyst

in our prior KOL checks. The press release noted that the safety results were consistent BofAS jason.gerberry@bofa.com

with the profile established in achondroplasia (ACH) setting, with no new safety signals

Chi M. Fong

observed. Next steps include: 1) regulatory filings in global markets, starting with the Research Analyst

FDA in 3Q, 2) full data presentation at an upcoming medical meeting. We view the BofAS chi.fong@bofa.com

update positively, as label expansion in HCH (estimated 14k TAPP) could help offset

Dina Ramadane

Voxzogo’s competitive headwinds in ACH. That said, we model relatively conservative Research Analyst

~$250m peak for Vox HCH given emerging competitors in the space, including BofASdina.ramadane@bofa.com

infigratinib Ph2 readout in 2H. We maintain Buy on underappreciated growth and Richard Wagner >>

durability of BMRN’s portfolio assets. Research Analyst

MLI (UK)

richard.wagneriii@bofa.com

Melanie Fong

Research Analyst

BofAS

melanie.fong@bofa.com

Stock Data

Price 50.20 USD

Price Objective 80.00 USD

Date Established 18-May-2026

Investment Opinion B-1-9

52-Week Range 49.26 USD - 66.28 USD

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Free Float 99.4%