BioMarin Announces New and Updated Data at 2021 American College of Medical

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Normal Progression of Bone Age with Chronological Age Over 60 Months with Vosoritide for Children with Achondroplasia

Vosoritide Well-Tolerated, Safety Profile Unchanged

Data on Quality of Life, Natural History, and Caregiver Experience Provide Additional Insights into Lifetime Impact of Achondroplasia

SAN RAFAEL, Calif., April 15, 2021 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) presented new and updated data at the 2021 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting that demonstrates the Company’s ongoing commitment to understanding the lifetime impact of achondroplasia and the potential of an investigational treatment choice to address the root cause of achondroplasia.

BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

BioMarin Pharmaceutical logo (PRNewsfoto/BioMarin Pharmaceutical Inc.)

The Company provided an update on its investigational treatment, vosoritide, an analog of C-type Natriuretic Peptide (CNP) in children with achondroplasia, the most common form of disproportionate short stature in humans. An ongoing, open-label, Phase 2 extension study of vosoritide for achondroplasia showed that improvement in growth velocity is sustained over 5 years of treatment and does not reduce the total duration of the growth period. Bone age progressed normally and posterior-anterior (PA) X-rays of the hand annually showed no significant changes in bone mineral content or bone mineral density.

The mean (±SD) increase in AGV observed over 60 months of treatment was 1.35 (±1.07) cm/year. There was an overall mean (±SD) increase in height Z-score (which measures the height deficit in standard deviations relative to the mean for age and gender-matched average stature children) at 60 months of 0.78 (±0.70) using the CDC standards for average stature children. Vosoritide was well tolerated at the doses of 15 and 30 µg/kg/day, and the safety profile remained unchanged with no new types of adverse events (AEs) developing over time, and no serious AEs were related to therapy.

“We are encouraged by the new data emerging from this extension study indicating that after five years of treatment, bone age is not accelerated and the total duration of the growth period is not shortened,” said Paul Harmatz, M.D., Professor in Pediatrics with a focus on skeletal dyplasias at UCSF Benioff Children’s Hospital in Oakland, California and clinical investigator in the vosoritide clinical program. “While still early, we’re seeing that proportionality is not worsening. We look forward to further follow up over time in the extension study for additional insights on proportionality.”

“It is important to understand the impact of achondroplasia on those affected and their caregivers in order to develop a therapeutic choice for families that has the potential to alter the course of achondroplasia in a way that is considered meaningful by families and treating physicians,” said Hank Fuchs, M.D., President Worldwide Research and Development. “We are…



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