BioMarin's dwarfism drug Voxzogo scores European nod for young children, awaits looming FDA decision

BioMarin building
BioMarin's Voxzogo now awaits a scheduled FDA decision this November. (BioMarin)

Voxzogo is just one of two medicines BioMarin executives think could completely transform the biotech. And now, with an approval in Europe, the achondroplasia treatment may be well on its way. 

The European Commission on Friday approved the once-daily injection to treat achondroplasia, the most common form of skeletal dysplasia that leads to dwarfism, BioMarin announced. Voxzogo, which is also awaiting an FDA decision this November, is now the first approved achondroplasia treatment in Europe.

France on Friday said it would move to grant a temporary authorization so patients can access Voxzogo immediately, BioMarin said. The company will price the treatment at roughly $300,000 per year, excluding discounts. Germany's initial list price will likely be similar, the company noted. 

BioMarin is prepared to launch the drug within four to six weeks of approval, the company said in its second quarter earnings call. 

RELATED: BioMarin gears up for two 'transformational' launches, Voxzogo and Roctavian: CEO

With the European go-ahead, the injection can be used in children from the age of 2 until their growth plates close, which occurs after puberty, BioMarin explained. 

The decision turned out to include children younger than what analysts had previously predicted, given the company presented data for kids only as young as five. This could help BioMarin achieve its sales goals more quickly, the SVP Leerink analysts said. 

BioMarin sees Voxzogo as a potential blockbuster, raking in over $1 billion in sales annually, although some analysts have placed it closer to $600 million by 2025. 

It likely won’t be difficult to locate potential Voxzogo patients given that many of them have been diagnosed at birth, according to SVP Leerink. 

There are about 11,000 children across Europe, the Middle East and Africa with achondroplasia and could be eligible for Voxzogo, BioMarin estimates. Around a third of those children live in countries under the EU’s decision on Friday. 

The EC’s decision bodes well for BioMarin’s expectation that Voxzogo, alongside its hemophilia A gene therapy Roctavian, will be “transformational” for the San Rafael, California-based biotech, according to CEO J.J. Bienaimé.

The drug could end up BioMarin’s “largest brand yet,” chief commercial officer Jeff Ajer told analysts earlier this month.

RELATED: BioMarin's dwarfism drug clears phase 3, poised for 2021 launch

Voxzogo, also known as vosoritide, is a modified C-type natriuretic peptide (CNP) that promotes endochondral bone growth, the process when growing cartilage is replaced with bone to form a growing skeleton. The treatment is designed to target the underlying cause of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling, BioMarin has explained. 

The EC’s decision comes just two months after the injectable scored a European Medicines Agency recommendation in late June. It was based on data from BioMarin’s late-stage trials, which found that Voxzogo helped children living with achondroplasia grow more quickly compared with those on placebo. 

In the 121-patient study, Voxzogo helped improve the growth velocity for children aged 5 to 14 from baseline by roughly 1.57 cm. 

People living with achondroplasia can also face serious health issues such as sleep apnea or spinal stenosis, in which the narrowing of the spine puts pressure on spinal nerves. Some patients may need surgery to reduce pressure on those nerves or to straighten bowed legs.