BioMarin to present new VOXZOGO imaging data and sNDA for hypochondroplasia at ASBMR
BioMarin Pharmaceutical will showcase results from 13 studies at the 2026 American Society for Bone and Mineral Research meeting, including post‑hoc imaging analysis of VOXZOGO in children with achondroplasia. The company has filed a supplemental New Drug Application to seek approval of VOXZOGO for hypochondroplasia, targeting a 2027 launch if the regulator approves. VOXZOGO is already approved for achondroplasia in the United States, Japan and Australia.
Why it matters
The sNDA could add a new indication and expand the market for VOXZOGO, potentially increasing future revenues if approved for hypochondroplasia. The imaging data may strengthen the case for early treatment, influencing clinician adoption.
Key facts
- 1BioMarin will present data from 13 studies at the ASBMR 2026 Annual Meeting. prnewswire.com
- 2The presentations include new imaging results from a post‑hoc analysis of a randomized, double‑blind, placebo‑controlled trial of VOXZOGO on foramen magnum and craniocervical junction growth in children with achondroplasia, including infants. prnewswire.com
- 3BioMarin recently submitted a supplemental New Drug Application to the FDA for VOXZOGO to treat hypochondroplasia. prnewswire.com
- 4The company expects a potential 2027 launch of VOXZOGO for hypochondroplasia if the sNDA is approved. prnewswire.com
- 5VOXZOGO is currently approved in the United States, Japan and Australia for achondroplasia. investing.com
- 6BioMarin estimates about 14,000 children worldwide could be eligible for VOXZOGO treatment for hypochondroplasia. investing.com
Summary written by AlphAI AI Desk from 2 of 2 sources. Not investment advice. Figures are as stated by the linked sources.