The success of Voxzogo is creating problems for BioMarin. With the launch going “way, way better” than expected, access to fill-finish capacity has emerged as a bottleneck that is constraining the rate of growth in 2023.
BioMarin won FDA approval for Voxzogo in achondroplasia, a form of dwarfism, in late 2021 and landed the runner-up spot in last year’s #FierceMadness drug name tournament championship. The product has emerged as a growth driver, topping sales forecasts to take the heat off the faltering European launch of BioMarin’s hemophilia A gene therapy Roctavian.
The good news continued for Voxzogo in the second quarter, but there was a sting in the tail. Sales of the rare disease drug jumped 229% to $113.3 million in the three months up to June 2023, but supply issues have put a ceiling on how big the product can grow in the near term.
“Supply remains tight from an inventory perspective,” Greg Guyer, Ph.D., chief technical officer, manufacturing and technical operations at BioMarin, told investors on the second-quarter results conference call. “My team is doing everything we can to continue to escalate or accelerate supply from the CMO. There's no issue, it's just trying to accelerate the supply availability faster than what we had planned.”
The situation informed BioMarin’s outlook for the rest of the year. Buoyed by its performance over the first six months of 2023, the biotech raised its forecast for Voxzogo for the full year—but it would have bumped the outlook higher still if it had unconstrained supply. In the end, BioMarin settled on raising its Voxzogo sales forecast to the $400 million to $440 million range.
BioMarin added $20 million to the bottom end of its range, but only raised the top end by $10 million as it bumped up against the ceiling created by the availability of fill-finish capacity. The biotech makes the drug substance itself—and has capacity to support blockbuster sales—but relies on a third party for the final steps.
“There's a lot of lead time when you want to increase the volume,” BioMarin CEO Jean-Jacques Bienaimé said on the call with investors. “That's what we are facing here because Voxzogo is doing way, way better than anybody, including ourselves, anticipated in terms of penetration.”
Bienaimé expects the supply problems to ease up next year and to be resolved by 2025. Even with the issue persisting into next year, the CEO believes the supply plan supports revenues that would “easily beat the current consensus for Voxzogo in ‘24.”