Please ensure Javascript is enabled for purposes of website accessibility

BioMarin to Present Results of Successful Achondroplasia Study

By Cory Renauer - Sep 11, 2020 at 12:54PM

You’re reading a free article with opinions that may differ from The Motley Fool’s Premium Investing Services. Become a Motley Fool member today to get instant access to our top analyst recommendations, in-depth research, investing resources, and more. Learn More

Daily injections of vosoritide sped up growth rates for children with the most common form of dwarfism.

Rare disease drug specialist BioMarin (BMRN -0.97%) recently shared successful top-line results from a clinical trial of vosoritide, an injected treatment intended to increase growth rates for children born with achondroplasia. On Saturday, the company will present results from the pivotal study at this year's meeting of the American Society for Bone and Mineral Research (ASMBR) in a virtual oral presentation to take place at 11:50 a.m. EDT.

Achondroplasia occurs in between 1 in 15,000 and 1 in 40,000 newborns. This makes the hereditary condition the most common form of short-limbed dwarfism, and there are currently no effective treatment options for it on the market. Those born with achondroplasia have trouble converting cartilage into bone, particularly in the arms and legs. While shorter stature is the most obvious symptom, complications of the condition can also include life-threatening spine malformation and chronic pain. 

Physicians at a medical conference.

Image source: Getty Images.

During the trial, children treated with daily injections of vosoritide grew significantly faster than those receiving a placebo. At the 52 week check-in, the average growth velocity of children given vosoritide was 0.62 inches per year faster than the placebo group.

The European Medicines Agency began reviewing a marketing application for vosoritide on Aug. 13, and BioMarin submitted a new drug application to the FDA on Aug. 20. The FDA has until Oct. 19 to request more information from BioMarin or begin its review process, which generally takes 10 months to complete.

Cory Renauer has no position in any of the stocks mentioned. The Motley Fool recommends BioMarin Pharmaceutical. The Motley Fool has a disclosure policy.

Invest Smarter with The Motley Fool

Join Over 1 Million Premium Members Receiving…

  • New Stock Picks Each Month
  • Detailed Analysis of Companies
  • Model Portfolios
  • Live Streaming During Market Hours
  • And Much More
Get Started Now

Stocks Mentioned

BioMarin Pharmaceutical Inc. Stock Quote
BioMarin Pharmaceutical Inc.
$95.07 (-0.97%) $0.93

*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

Related Articles

Motley Fool Returns

Motley Fool Stock Advisor

Market-beating stocks from our award-winning analyst team.

Stock Advisor Returns
S&P 500 Returns

Calculated by average return of all stock recommendations since inception of the Stock Advisor service in February of 2002. Returns as of 08/11/2022.

Discounted offers are only available to new members. Stock Advisor list price is $199 per year.

Premium Investing Services

Invest better with The Motley Fool. Get stock recommendations, portfolio guidance, and more from The Motley Fool's premium services.