- A drug targeting the most common cause of dwarfism succeeded in a late-stage study, positioning developer BioMarin Pharmaceutical to soon seek regulatory approval for what would be the first medicine for a bone growth disorder called achondroplasia.
- While BioMarin did not disclose many details, the company said its drug vosoritide helped children's bones grow faster than did placebo after one of year treatment. Most side effects were mild, and no serious adverse events were judged to be related to vosoritide, BioMarin said.
- Helping people of disproportionate short stature grow taller is a controversial proposition, but vosoritide could help those with achondroplasia avoid some of the many health complications that result from the disorder, which is spurred by a genetic mutation.
BioMarin has built its business around developing drugs for rare genetic diseases. It's had substantial success, winning approval in the U.S. or Europe for seven medicines in its two-decade history as a company.
The next 12 to 18 months could see BioMarin add two more to its portfolio, with regulatory applications expected for vosoritide and the biotech's hemophilia gene therapy known as valrox.
BioMarin will hold discussions with regulators on filing vosoritide in the first half of next year, the company said Monday when announcing positive study results for the drug.
Success in Phase 3 for vosoritide comes roughly seven years after BioMarin first began testing the injection as a potential treatment for people with achondroplasia. If approved, vosoritide would become the first therapy to treat the underlying cause.
About 1 in 25,000 babies are born with achondroplasia, which stems from an error in a gene that regulates bone growth and accounts for most cases of dwarfism. Meaning "without cartilage formation," achondroplasia disrupts a process by which cartilage is converted to bone in the arms and legs.
The average height of people with achondroplasia is between 4 feet 4 inches and 4 feet 1 inch depending on gender.
In BioMarin's trial, which enrolled 121 children between ages five and 14, treatment with vosoritide led to a 1.6 centimeter per year increase from baseline in growth velocity after adjusting for placebo results. That largely met or surpassed expectations among Wall Street analysts who follow BioMarin, but is lower than the 2 centimeter mark BioMarin showed in a Phase 2 study across roughly one-tenth as many patients.
Still, the data should position the company well for potentially gaining Food and Drug Administration approval, analysts from SVB Leerink, Piper Jaffray, Cantor Fitzgerald and Stifel wrote Monday.
Beyond that headline number, however, details on the data were scant. BioMarin said it would present further results at an upcoming medical meeting, which would provide a better opportunity to gauge how vosoritide impacts bone growth and health, as well as patient quality of life.
Another question is how BioMarin defines "clinically significant" blood pressure decreases, one potential safety concern. No such declines were observed in the Phase 3 test, the company said.
vosoritide, given by injection daily, is intended only for children whose growth plates are still "open," which accounts for approximately a quarter of the 22,000 people living with achondroplasia.
The drug is a form of C-type natriuretic peptide that BioMarin designed to last longer in the body to help boost its therapeutic effect.
In a recent presentation, BioMarin said it expects to launch vosoritide in 2021, pending approvals. Potentially closer is a launch for valrox, which it plans to submit this year.