What's happening: BioMarin Pharmaceutical (NASDAQ:BMRN) shares increased by 10% earlier today following positive midstage trial results being released for its BMN-111, a treatment for children with achondroplasia -- the most common form of human dwarfism.
Why It's happening: BioMarin Pharmaceutical is one of the most successful developers of therapies for rare disease, and the results from this trial suggest that BMN-111 could end up being yet another important win for the company.
BMN-111 is a stabilized formulation of C-type natriuretic peptide, a naturally occurring human peptide that helps promote bone growth.
In this phase 2 study, 10 achondroplasia patients taking a 15 microgram per kilogram dose per day of BMN-111 showed an average increase of 50% in annualized growth velocity versus their prior six month annualized growth velocity. That's enough of an improvement, if it's maintained, for BioMarin to suggest that BMN-111 could allow the roughly 24,000 patients under 18 with this condition to resume a normalized growth rate.
BioMarin plans to sit down with regulators and create a plan to move the 15 microgram dose of BMN-111 into phase 3 trials that, if successful, could lead to an eventual FDA approval. BioMarin also intends to explore whether or not a higher dose of BMN-111 could enable "catch-up" growth in the event of delayed treatment.
Todd Campbell owns shares of BioMarin Pharmaceutical. Todd owns the equity research firm E.B. Capital Markets, LLC. E.B. Capital's clients may have positions in the companies mentioned. The Motley Fool recommends BioMarin Pharmaceutical. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.