BioMarin Pharmaceutical presented new long‑term data on its approved therapy VOXZOGO (vosoritide) at the Pediatric Endocrine Society’s 2026 Annual Meeting in San Francisco, held from April 30 to May 3, 2026. The presentation highlighted that VOXZOGO treatment led to significant improvements in arm span, bone density, and height in children with achondroplasia, with arm span Z‑scores improving across all age groups and a mean height increase of 10.60 cm after six years of treatment for those who started therapy after age 5.
The achondroplasia data also showed that the arm span‑to‑height ratio remained stable, indicating proportional skeletal growth. These findings reinforce VOXZOGO’s clinical benefit profile beyond height, supporting its continued use as the standard of care for achondroplasia and expanding its potential indication to hypochondroplasia.
BioMarin also announced that data from two ongoing studies on VOXZOGO in hypochondroplasia would be presented at the same meeting. A single‑arm Phase 2 study from Children’s National Hospital reported a statistically significant increase in total body minus head bone mineral density of 0.03 g/cm² and a 54.84 g rise in bone mineral content after 12 months of therapy. BioMarin plans to share topline results from its registration‑enabling Phase 3 pivotal trial (CANOPY‑HCH‑3) in the first half of 2026, with a potential regulatory submission later that year.
The upcoming CANOPY‑HCH‑3 topline results represent a key near‑term catalyst for the company, as a positive outcome could expand VOXZOGO’s approved indications to hypochondroplasia, a condition that currently lacks approved therapies. A regulatory approval would broaden the drug’s addressable market and strengthen its competitive position in the skeletal‑conditions franchise.
In the broader business context, BioMarin’s Q4 2025 revenues reached $875 million, a 17% year‑over‑year increase largely driven by VOXZOGO, while full‑year 2025 revenues totaled $3.22 billion, up 13% YoY. VOXZOGO contributed 26% year‑over‑year growth. The company also announced the acquisition of Amicus Therapeutics in December 2025, expected to close in Q2 2026, which is poised to accelerate and diversify revenue streams with products such as Galafold and Pombiliti + Opfolda.
Management emphasized the significance of the data. Dr. Greg Friberg, Executive Vice President and Chief Research & Development Officer, said, “We are committed to understanding and sharing the clear impact of long‑term treatment with VOXZOGO on health measures beyond height, including arm span and bone density, that are meaningful to the thousands of children around the world receiving this medicine. With more than ten years of clinical research now, we have seen again and again a breadth of data that continue to demonstrate that long‑term and early treatment are critical to ensure the greatest benefit in children with achondroplasia.” CEO Alexander Hardy added, “The Amicus transaction, which is expected to close in the second quarter, represents a compelling opportunity to reach more patients around the world and further strengthen our revenue growth through the next decade.” Pediatric endocrinologist Bradley Miller noted, “With VOXZOGO, we now have a depth and duration of evidence that is unmatched in the treatment of achondroplasia — providing meaningful insight not only into growth, but into the broader, sustained impact on a child’s health.”
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