BioMarin's dwarfism drug Voxzogo could be its next blockbuster, but ethics questions loom over the launch

BioMarin
BioMarin's achondroplasia medicine Voxzogo scored an FDA approval. The drug should be available next month, the company says. (BioMarin)

BioMarin has won the FDA's go-ahead for its next big launch. But the drug's arrival may be unwelcome news for those who've built a community out of living full lives with dwarfism.

The FDA approved Voxzogo, also known as vosoritide, for a form of dwarfism called achondroplasia. With the FDA nod, Voxzogo becomes the first treatment licensed in the U.S. for achondroplasia.

After conducting a recent doctor survey about the condition, SVB Leerink analyst Joseph Schwartz recently wrote to clients that he sees a "significant opportunity that could revive growth that has been stagnant" for BioMarin.

The FDA approved the medicine, a daily injection, to increase linear growth in people who have achondroplasia and are five years and older with open growth plates, which typically close around age 18.

People with the achondroplasia, the most prevalent form of skeletal dysplasia, develop a "short stature and truncated limbs," SVB's Schwartz wrote in a note last weekend. For FDA approval, BioMarin submitted data showing that people on the drug grew faster than those on placebo in a year-long study.

The company expects the medicine to be available in mid- to late-December and will begin promoting it immediately.

While investors have been somewhat skeptical of the medicine's commercial potential, BioMarin execs have voiced enthusiasm. This summer, chief commercial officer Jeff Ajer said the company had teams "in place and well-prepared for what could be BioMarin's largest brand yet." For his part, SVB Leerink's Schwartz and his team are projecting $1 billion in peak sales.

And while BioMarin is celebrating the FDA approval, the arrival of Voxzogo may be unwelcome news for some in the achondroplasia community, Stat News reports. Groups such as the Little People of America have worked hard to communicate the message that people with achondroplasia can live full lives, and they see the new medicine as a threat to their work, according to the publication. 

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Voxzogo, a first-in-class C-type natriuretic peptide analog, works by regulating the signaling pathway downstream of the fibroblast growth factor receptor 3 gene to assist bone growth, BioMarin said. In people with achondroplasia, a mutation in the gene hinders growth.

"Voxzogo is a medical first that is rooted in BioMarin's focus on molecular genetics and targets the underlying cause of the condition," BioMarin CEO Jean-Jacques Bienaimé said in a Friday statement. "More than a decade of scientific research underpins the medical advance that Voxzogo represents."

The Voxzogo approval came via FDA's accelerated pathway, so BioMarin is meant to prove the drug's benefits in confirmatory trials to convert the regulatory nod to a full approval. To meet its accelerated approval requirements, BioMarin plans to keep an open-label study running to compare results with the natural course of achondroplasia.

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

BioMarin also won a Rare Pediatric Disease Priority Review Voucher with its Voxzogo approval. 

Voxzogo is one of two "transformational" medicines that BioMarin has been gearing up to launch, CEO Bienaimé said this summer. The other, hemophilia A gene therapy Roctavian, suffered an FDA rejection last summer. BioMarin hopes to resubmit the drug to the FDA next year.

Aside from Voxzogo, other potential achondroplasia treatments are in development at Ascendis Pharma, BridgeBio and Pfizer, Schwartz noted.