JPM24: BioMarin's new CEO Hardy says top priority is expansion of dwarfism drug Voxzogo

In his first presentation as the new CEO of BioMarin, Alexander Hardy left no doubt about his mission for the rare and genetic disease specialist.

The top priority for the former Genentech CEO is to accelerate and maximize the commercial potential of dwarfism drug Voxzogo, Hardy said Tuesday at the J.P. Morgan Healthcare Conference. His second objective is to ramp up the commercialization of Roctavian, a newly approved gene therapy for hemophilia A. Thirdly, it is up to Hardy to identify BioMarin’s most promising R&D projects and make tough decisions about what to cut.

Given his experience heading up Roche subsidiary Genentech for the last five years, Hardy is equipped to take on these challenges. During his time in charge, Genentech launched 10 medicines, including blockbuster macular degeneration drug Vabysmo. The company’s revenue jumped 24% and did so efficiently as profit-per-employee grew by 69%.

“Why did I choose BioMarin? I think it’s an enormously exciting company, in a really interesting point in its development,” Hardy said. “I think there’s tremendous opportunities from a value-creation and impact-patient standpoint.”

As for Voxzogo, which the company guided to generate between $435 million and $455 million in 2023, Hardy characterized the achondroplasia launch as having “tremendous momentum,” despite some supply issues that prevented BioMarin from meeting demand last year.

Hardy said that supply will be “unconstrained” by the middle of next year. After supplying 2,800 patients through its CDMO last year, total capacity bumps up to 6,000 patients this year and 8,000 in 2025.

“Looking forward, we believe we’ve got ample supply to be able to meet the growing demand,” Hardy said. 

Meanwhile, demand is increasing with an FDA label expansion three months ago to treat children of all ages with open growth plates, versus the drug's previous nod for children 5 and older.

The longer-term focus with Voxzogo is to bring several other pathway indications that could expand its treatable patient population from its present of 21,000 to 600,000.

With respect to Roctavian, which was approved by the FDA in June, Hardy said that the company reached an important milestone in dosing its first patient in the U.S. at the end of December, though the launch has been “slower than anybody would have expected or would have wanted.”

Hardy, who was instrumental in the successful launch of Genentech’s hemophilia A treatment Hemlibra, called Roctavian “highly disruptive.”

“We’ve made important progress in market access and readiness, but 2024 and 2025 are going to actually inform what that uptake curve is,” he added.

Besides the launch efforts, the new CEO will have difficult decisions to make as the company attempts to prioritize early-stage assets and cut its R&D expenses.

“We’re gonna be focusing absolutely on the science, the potential patient impact, but that’s highly related to the market potential and the return on investment,” Hardy said. “We’ve gotta be very disciplined through the early stages of research with our spend. We’ve gotta have high bars for success.”