BioMarin is facing challenges due to the overwhelming success of its drug, Voxzogo, used to treat achondroplasia, a type of dwarfism. The drug’s launch has far exceeded expectations, leading to a surge in demand. However, the company is struggling with access to fill-finish capacity, which is now limiting its growth potential in 2023.
Voxzogo received FDA approval for achondroplasia in late 2021 and has quickly become a significant driver of growth for BioMarin. The drug’s sales surpassed forecasts, alleviating some pressure caused by the slower European launch of BioMarin’s hemophilia A gene therapy, Roctavian.
In the second quarter of 2023, Voxzogo sales skyrocketed by 229% to reach $113.3 million. However, supply issues are currently hindering the product’s growth potential. BioMarin had to adjust its outlook for the year accordingly. Although the biotech raised its forecast for Voxzogo sales for the full year, the constrained supply limited the increase to a range of $400 million to $440 million.
“Supply remains tight from an inventory perspective. 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.”
– Greg Guyer, Ph.D., chief technical officer, manufacturing and technical operations at BioMarin
The bottleneck is primarily due to limited fill-finish capacity. While BioMarin can manufacture the drug substance in-house with the capacity to support blockbuster sales, the final fill-finish steps are outsourced to a third party. This has created a constraint on the drug’s overall growth.
BioMarin’s CEO, Jean-Jacques Bienaimé, believes that the supply problems will improve in the coming year and expects them to be resolved by 2025. Despite the current challenges, Bienaimé is confident that the supply plan will result in revenues that will surpass the current consensus for Voxzogo in 2024.
In summary, BioMarin’s Voxzogo has enjoyed tremendous success, but the supply chain bottleneck is limiting its growth potential in the near term. The company remains optimistic that the supply issues will be resolved in the coming years, enabling the drug to continue its impressive growth trajectory.
