BioMarin’s Dwarfism Drug Improves Growth in a Mid-Stage Study

BioMarin Pharma Issues Positive Update From Dose-Finding StudyBioMarin Pharmaceutical Inc. (NASDAQ/BMRN) shares soared at a near all-time high after the company announced positive results from a mid-stage study for its drug to treat the most common form of dwarfism.

The company’s shares rose 6.5% to $131.93 in pre-market trading on the morning of Thursday, June 18.

“We are very encouraged to have observed evidence of activity with vosoritide in children participating in our Phase II study,” said Wolfgang Dummer, Vice President of Clinical Development at BioMarin.

The drug is designed for patients who are suffering from medical conditions caused by slow growth. There are so many different reasons that can lead to dwarfism. though the illness is mainly caused by bone growth disorders, which can sometimes be treated by surgery.


The company said that the drug would increase 50% of patients’ annualized growth velocity compared to their annualized prior six-month natural history baseline growth velocity. (Source: BioMarin Pharmaceutical Inc., June 17, 2015.)

Dummer also added, “This increase in growth velocity, if maintained, could allow children with achondroplasia (dwarfism) to resume a normalized growth rate.”

Dwarfism is a genetic disorder that affects about 1 in 15,000-40,000 newborns. “We are looking forward to working with health authorities worldwide as we continue to develop vosoritide for patients with achondroplasia globally,” said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer at BioMarin.

“It is estimated that about 96,000 patients in our established territories are afflicted with achondroplasia, so approximately 25%, or 24,000, are under 18 years of age and in our addressable market.”

The development in the process of treatment for those suffering from dwarfism has advanced to the highest level so far. Analysts are expecting that BioMarin would have a broader market share in the next few years and that they could expect higher sales and earnings for the years to come.

On the other hand, analysts remain cautious prior to the Phase III approval from the Food and Drug Administration (FDA). But if approved in Phase III, they expect the stock could go significantly higher.