BioMarin Pharmaceutical Inc. (BMRN) said that children in the open-label long-term extension of the phase 3 study of vosoritide maintained an increase in annual growth velocity through the second year of continuous treatment. It is an investigational, once daily injection analog of C-type Natriuretic Peptide.
The company noted that children treated with vosoritide demonstrated cumulative height gain of 3.52 cm at year 2 compared to untreated children. It is is the sum of the first year (1.73) and the second year (1.79).
The company said that the 2-year data demonstrated that vosoritide, administered at 15ug/kg/day was generally well tolerated with no new safety findings. The majority of adverse events were mild and no serious adverse events were reported as study drug-related.
Injection site reactions were the most common drug-related AEs, and all were transient. No clinically significant blood pressure decreases or new safety findings were observed.
BioMarin said its marketing applications for vosoritide are currently under review by both the Food and Drug Administration and the European Medicines Agency, and if approved would be the first therapy for achondroplasia in the U.S. and Europe.
The FDA’s Prescription Drug User Fee Act or PDUFA target action date is August 20, 2021. The Committee for Medicinal Products for Human Use (CHMP) opinion is expected in Europe in the second half of 2021.
Vosoritide has also received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
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