After a period of "stagnant" growth at BioMarin—as characterized by one analyst team—the drugmaker could be on the brink of its next big launch. The company's dwarfism drug Voxzogo already has its European approval in hand, and it's due for an FDA decision later this week.
BioMarin management has voiced high expectations for Voxzogo, but investors haven't come around to the med, SVB Leerink analyst Joseph Schwartz wrote to clients on Sunday. After conducting a doctor survey on the medicine and others, the analyst sees "significant opportunity that could revive growth that has been stagnant" for BioMarin.
In August, Voxzogo scored approval in Europe to treat achondroplasia in patients two years and older until their growth plates close, which is typically around 18 years. The med is under review at the FDA, with a decision date set for Saturday, Nov. 20.
Patients with achondroplasia, the most prevalent form of skeletal dysplasia, develop a "short stature and truncated limbs," Schwartz wrote. The condition "may appear cosmetic on the surface," but it's "associated with life-threatening comorbidities that can increase mortality," he added. Doctors consistently see it as a disease with a "huge unmet need."
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After SVB Leerink's recent doctor survey, Schwartz and his team believe adoption "could be swift," with about 30% of eligible patients starting on the therapy within a year. The team is projecting $1 billion in peak sales.
One factor supporting uptake is that patients are "readily identifiable" and most are "born to parents with normal height who are likely more motivated than not to treat their children," the analyst noted. In addition, doctors may want to start treatment quickly while a patient's growth plates are open.
From a "commercial execution standpoint," the company has an "extensive sales force worldwide that could readily execute a global launch from their years of being a commercial company," Schwartz noted.
But the drug faces some uncertainties. The disease is "fraught with social complexities making the adoption of Voxzogo not very straightforward." With that challenge considered, and because some of other BioMarin launches haven't lived up to management's expectations, some investors remain skeptical, Schwartz wrote.
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With all of the factors considered, Schwartz believes there's reason to buy into BioMarin's enthusiasm for Voxzogo. This summer, BioMarin's chief commercial officer Jeff Ajer said the company had teams "in place and well-prepared for what could be BioMarin's largest brand yet."
Aside from BioMarin, other potential achondroplasia treatments are in development at Ascendis Pharma, BridgeBio and Pfizer, Schwartz wrote.