BioMarin Pharmaceutical (NASDAQ:BMRN) executives highlighted a three-pillar strategy focused on growth, innovation, and “value commitment” during a fireside chat hosted by Leerink Partners’ Joe Schwartz, pointing to recent revenue momentum, multiple clinical milestones, and the planned addition of Amicus assets as key components of the company’s next phase.
Strategy framed around growth, innovation, and value
Chief Financial Officer Brian Mueller said the company is “really excited about 2026” following what he described as a strong 2025. BioMarin reported total revenue growth of 13% in 2025, with its Skeletal Conditions unit (including VOXZOGO) growing 26% and Enzyme Therapies growing 9%.
On profitability and cash generation, Mueller said BioMarin has emphasized disciplined expense management and “efficient cost allocations” while growing the top line, which he characterized as leading to leveraged growth. He added that after adjusting for special items in the 2025 P&L, BioMarin expects earnings per share growth at the midpoint of guidance at roughly three times revenue growth, alongside a third year of operating margin expansion and cash flow growth.
VOXZOGO: durability, broader benefits, and next-generation program
Chief Commercial Officer Cristin Hubbard discussed how the company views “stickiness” in VOXZOGO as additional treatment options emerge for achondroplasia. She said BioMarin’s research and conversations with physicians and caregivers suggest switching is less likely when patients are doing well on therapy, noting that on average about two-thirds of key opinion leaders and caregiver research indicate the chance of switching is low in that situation. Hubbard said the company believes decision-making ultimately rests with parents and caregivers, with physicians typically providing options and factual information.
Hubbard also emphasized BioMarin’s focus on benefits beyond height, describing publications and longer-term data showing areas such as foramen magnum benefits, symptomatic spinal stenosis benefits, and statistically significant improvements in proportionality, including leg deformities such as tibial bowing. She also referenced quality-of-life improvements seen in longer-term data.
Mueller added that BioMarin has been developing VOXZOGO for more than 15 years and has patients who have been on therapy for 10 years. He said the drug was approved in 2021 under a conditional approval, and that BioMarin now has a long-term dataset following patients to final adult height. Mueller said the company plans to file that dataset this year, which could potentially support a label amendment.
On BMN 333, BioMarin’s long-acting CNP program for achondroplasia, management said the strategy is not purely convenience-driven. Mueller said BMN 333 is intended to be “superior to VOXZOGO and the competition” based on data seen so far. He described a phase II/III seamless design trial enrolling 120 patients, designed to detect superiority versus VOXZOGO at the 90% level and to assess both annualized growth velocity and benefits beyond height.
Hypochondroplasia data timing and diagnosis focus
BioMarin also discussed VOXZOGO’s potential expansion into hypochondroplasia. Hubbard said the company is “really excited” to see phase III data “soon,” with timing described as coming in the first half of 2026. She said BioMarin has “high confidence in the science” and pointed to strong signals from a sponsored trial by Dr. Dauber as part of the backdrop for expectations.
As an early indicator of interest, Hubbard cited the speed of enrollment in BioMarin’s phase III trial, which she said exceeded expectations and can serve as a proxy for excitement in the indication. However, she stressed that diagnosis and education are expected to be central challenges. Hubbard said BioMarin believes hypochondroplasia prevalence is similar to achondroplasia, but that the total addressable patient population appears lower because the condition is not diagnosed at the same rate and patients are often diagnosed around age five or six.
Amicus acquisition: expansion levers and accretion outlook
Turning to the planned Amicus deal, Hubbard said BioMarin sees opportunity for both geographic expansion and deeper penetration for Galafold, noting Amicus is in about 40 countries while BioMarin has a footprint of roughly 80. She said diagnosis is a major opportunity in Fabry disease and described BioMarin’s capabilities in driving diagnosis and supporting therapy adherence as relevant strengths.
Mueller added that, subject to closing, the company believes there is potential to unlock additional value over time in both Galafold and Pompe beyond what was modeled for the transaction. He reiterated prior commentary that the deal could be slightly dilutive in calendar 2026 due to interest expense and integration costs, but accretive over its first 12 months and “substantial accretion” starting the following year, based on BioMarin’s deal model.
For Pompe, Hubbard said the product is currently reimbursed in 15 countries and suggested BioMarin’s larger footprint could support expansion. She also highlighted the importance of publishing real-world evidence to demonstrate value in patients switching from other enzyme therapies and said BioMarin would look to continue that work. On manufacturing strategy for Pompe, Mueller said BioMarin would need to wait until after closing to discuss details, while reiterating BioMarin’s view of itself as a “world-class” manufacturing organization.
Pipeline updates: Duchenne and ENPP1 deficiency
BioMarin provided updates on BMN 351 in Duchenne muscular dystrophy. Mueller said the program has three doses—6, 9, and 12 mg—and that BioMarin previously reported 26-week data from the 9 mg cohort showing mean dystrophin expression of 5%. He said the next key data points will involve higher-dose milestones, and that BioMarin will evaluate clinical benefit measures including the North Star Ambulatory Assessment and forced vital capacity, looking for correlation between dystrophin expression and clinical outcomes. Mueller said the company is aiming for 10% dystrophin expression as part of its decision-making, while emphasizing it will consider the “totality of the data.”
On BMN 401 for ENPP1 deficiency—an asset acquired through BioMarin’s Inozyme acquisition—Hubbard said the company is looking forward to pediatric data expected in the first half. She estimated a total addressable patient population of roughly 2,000 to 2,500 patients worldwide, while emphasizing that diagnosis and identifying patients will be a key focus. Mueller added that ultra-rare disease prevalence estimates can expand once a therapy exists, citing NAGLAZYME as an example where BioMarin previously cited global prevalence of about 1,100 patients but now has nearly 150% of that number on commercial therapy.
Finally, Mueller addressed VOXZOGO revenue seasonality, saying the company expects revenues to be weighted toward the second half of the year and particularly the fourth quarter due to international ordering patterns. He said roughly 75% of VOXZOGO revenue comes from outside the U.S., largely through single national payer systems whose procurement and budgeting contribute to back-half ordering, even as BioMarin expects to continue adding patients steadily quarter after quarter.
About BioMarin Pharmaceutical (NASDAQ:BMRN)
BioMarin Pharmaceutical Inc is a biopharmaceutical company specializing in the development and commercialization of therapies for rare genetic and metabolic diseases. The company focuses on addressing unmet medical needs by leveraging enzyme replacement therapy, small molecule pharmacological chaperones and gene therapy technologies. Headquartered in Novato, California, BioMarin operates research and development facilities in the United States and Europe.
The company’s commercial portfolio includes several approved therapies targeting inherited disorders.
