Biomarin’s Voxzogo disappoints in youngsters

The new achondroplasia treatment Voxzogo is a key part of Biomarin’s quest to become profitable this year and beyond. But the therapy’s chances of expanding into young children look to be on shaky ground after an update yesterday from a phase 2 study in patients up to five years old. Biomarin, without disclosing actual data, said there was merely a “trend” towards improvement with Voxzogo versus placebo, suggesting that statistical significance had not been hit. And the group’s president of worldwide R&D, Henry Fuchs, admitted during its fourth-quarter earnings call: “We didn't get a gigantic signal of effectiveness.” He pointed to the small trial size and “high variability” between patients. Adverse events might also have spooked investors: there was a case of sudden infant death syndrome, albeit deemed unrelated to therapy, and a “small increase” in sleep apnoea events that Biomarin said were mild to moderate. Overall, though, serious adverse events were higher in the placebo than the Voxzogo group. Investors are also wary about the approval chances of Biomarin’s haemophilia A gene therapy valrox, which is heading back to regulators backed by two-year data from the pivotal GENEr8-1 trial. Shares opened down 8%.

Biomarin's big hopes
Project Status 2022 guidance ($m) 2026e sales ($m)
Voxzogo  FDA accelerated approval Nov 2021 in achondroplasia pts aged ≥5 90-115 644
Valoctocogene roxaparvovec (valrox/Roctavian) FDA filing planned in June; CHMP opinion expected Q2 2022 - 635
Source: Evaluate Pharma & company communications.

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