- BioMarin Pharmaceutical can sell its drug for dwarfism in Europe after regulators there granted a market authorization, two months following the European Medicines Agency's endorsement of the California biotech company's treatment.
- BioMarin's drug, which it will sell under the brand name Voxzogo, is the first medicine to be made available in Europe for achondroplasia, the most common cause of dwarfism. About 4,000 children have the condition in the EU countries covered by the European Commission's licensure, BioMarin estimates.
- As with many rare disease treatments, Voxzogo will be extremely costly. Under an access plan with authorities in France, BioMarin plans to charge roughly $300,000 per patient per year. The drugmaker expects the initial price of the drug in Germany will be similar, although the company expects to make "material discounts" in one to two years as reimbursement agreements with individual country governments are negotiated.
Voxzogo is BioMarin's seventh drug approved by regulators in either the U.S. or Europe, the product of nearly two-and-a-half decades' worth of research into rare diseases.
Seven of those years have been spent testing Voxzogo, or vosoritide, in clinical trials for achondroplasia, which is often caused by a genetic mutation that impedes bone formation and leads to disproportionate short stature.
In a Phase 3 study, treatment with Voxzogo led to faster bone growth than did placebo over a year, a benefit that built on previous findings from a mid-stage trial. Most side effects were mild in nature and no severe adverse events related to Voxzogo were reported by BioMarin.
The European Commission, following the EMA's June recommendation, authorized Voxzogo for use in children from two years old until their growth plates are closed, which happens after puberty.
Regulators in the U.S., however, have sought two-year results from that Phase 3 study, which BioMarin submitted in the first quarter. As a result, the Food and Drug Administration extended its review of the company's application by three months, setting a decision deadline of Nov. 20.
A U.S. clearance would be another milestone for BioMarin, which is also returning to regulators for a second attempt at approval of its gene therapy for hemophilia A. The FDA, seeking more data, unexpectedly rejected the treatment last August and BioMarin later withdrew its application to the EMA as well.
BioMarin has since resubmitted to the EMA, which is expected to make a decision on the therapy by the second quarter of next year. That's also around when BioMarin anticipates re-filing an application with the FDA, pending positive two-year data from a Phase 3 study of the treatment.