BioMarin Pharmaceutical (NASDAQ:BMRN) executives highlighted 2025 revenue growth, improving profitability leverage, and a slate of clinical and commercial catalysts expected in 2026 during a discussion at a Barclays event featuring CFO Brian and Chief Commercial Officer Cristin.
2025 performance and profitability leverage
Management said the company exited what it characterized as a “very successful 2025,” reporting 13% total revenue growth for the full year. Within that, the company cited 26% revenue growth for Voxzogo and 9% growth for its enzyme therapies business unit.
Amicus acquisition: timing, scale, and integration focus
The executives pointed to the announced acquisition of Amicus as a key momentum item exiting 2025. The deal was announced in December, and the company said it expects to close in Q2, subject to customary closing conditions. If completed, management said the transaction would add two “high growth” approved rare disease assets to BioMarin’s portfolio, which it described as more than $3 billion of revenue from eight rare disease products.
BioMarin also noted that Amicus’s products generated over $600 million in 2025 revenues, and management indicated it will update guidance after the deal closes. The CFO discussed margin considerations, stating that Galafold is a small molecule with a “slightly higher margin,” while the Pombiliti plus Opfolda combination is “slightly lower” margin due in part to including a biologic component. He also referenced familiarity with cost-of-goods initiatives and manufacturing process improvements Amicus has been pursuing, which BioMarin plans to evaluate post-close.
On operating expenses, the CFO said BioMarin intends to “preserve and grow” Amicus’s existing commercial capabilities—particularly given Amicus’s experience in “switch” markets—while also leveraging BioMarin’s global infrastructure. He added that some expansion efforts may require incremental investment, but the company expects “operational and operational expense synergies,” with more detail to come after closing and integration planning.
Cristin said BioMarin’s near-term priority is to “do no harm” and sustain the current trajectory of the acquired medicines, while seeking to “inflect that curve” by increasing penetration and expanding geographic reach. She noted that Galafold is in 40 countries and Pombiliti is in 15, while BioMarin has a footprint of nearly 80 countries. Teams are evaluating which additional markets to enter first and the cadence of expansion.
Voxzogo outlook: guidance drivers and competitive context
For 2026, management said it expects both core business units—enzyme therapies and skeletal conditions—to grow in the high single digits, citing 7%–8% growth for each. The CFO also flagged a roughly 3% headwind to total revenue from declining royalty and other revenue, emphasizing that the key growth drivers remain the two business units.
For Voxzogo specifically, BioMarin guided to $975 million to $1.025 billion in 2026 revenue, with the midpoint implying approximately $1 billion. Management highlighted two primary swing factors for the range:
- International market access negotiations: the low end of guidance assumes potential price discounts in certain markets to open access to more patients over time; the high end assumes more favorable pricing outcomes.
- Competition-driven switching dynamics: the low end assumes more switching earlier; the high end assumes less switching later.
In discussing competition, the CFO emphasized that about 25% of Voxzogo revenue is in the U.S. and is the portion most directly exposed to the competitive dynamic discussed, which he framed as approximately 7% of BioMarin’s total revenue. He added that even differing assumptions around share loss fit within the $50 million guidance band and said the factors are not “largely material” relative to a portfolio he described as $3.3–$3.4 billion.
BioMarin also reiterated its view that it is well positioned competitively due to its established presence in the achondroplasia community and physicians’ familiarity with the product. Management said it expects to retain the infant label for ages 0–2, describing early initiation as “most important” and referencing international treatment guidelines.
Pipeline catalysts: Phase III readouts and next-stage programs
Executives outlined several 2026 pipeline catalysts, including two Phase III readouts in the first half of the year:
- Voxzogo in hypochondroplasia: a Phase III study is expected to read out in the first half of the year. Management described hypochondroplasia as similar to achondroplasia and referenced proof-of-concept data from an investigator-sponsored study.
- BMN 401 for ENPP1 deficiency: BioMarin said this enzyme replacement therapy—acquired via the Inozyme acquisition in 2025—also has Phase III data expected in the first half of the year.
On hypochondroplasia, management said the condition may be genetically as prevalent as achondroplasia, but the diagnosis rate is much lower—estimated at about 30%. BioMarin cited a total addressable patient population of around 14,000 for hypochondroplasia and said commercial planning is focused on disease awareness and shortening the diagnostic pathway, noting that children are currently diagnosed around age five to six. If approved, management said Voxzogo would be the only treatment for hypochondroplasia, and Cristin suggested early adoption could be fastest at centers with key opinion leaders or clinical trial involvement, while broader uptake may follow a “normal launch curve” given patients are dispersed across pediatric and endocrinology settings.
For BMN 401 in ENPP1 deficiency, Cristin said the Phase III program will assess normalization of pyrophosphate levels and a radiographic functional endpoint in pediatric patients. She said there are no current treatments and estimated a 2,000–2,500 global addressable patient population, while noting prevalence is unclear in publications and diagnosis may increase if an effective therapy becomes available.
BioMarin also discussed two internal clinical-stage programs:
- BMN 351 (Duchenne muscular dystrophy): the company is testing three dose levels and reported that at a middle dose it achieved 5% dystrophin expression at 25 weeks, which it described as roughly halfway to the expected steady-state timeframe (52 weeks or more). Management said this supports its aim of reaching 10% dystrophin expression and plans to share results from the highest 12 mg dose later in the year.
- BMN 333 (long-acting CNP): management described this as a potentially weekly injection and said the Phase II/III program is being designed not just for convenience but to be superior in efficacy in a head-to-head study versus Voxzogo and potentially versus competitors.
Separately, management referenced the broader CANOPY Phase II trials across additional growth-related conditions (ISS, Noonan’s, Turner’s, and SHOX). The CFO cited a combined “bullet” patient population size of 385,000 across those four conditions, while emphasizing BioMarin would expect to treat only a “very, very small fraction” of the most severe patients. He said the trials are enrolling and that BioMarin expects data next year.
Commercial updates: Palynziq label expansion and quarterly dynamics
The company discussed a recent Palynziq adolescent label expansion for patients aged 12 to 17. Cristin emphasized the potential quality-of-life benefit of lowering phenylalanine (Phe) levels to within the normal range and enabling a normal diet. She said BioMarin estimates about 1,500 eligible U.S. adolescent patients, and noted that more adolescents are in clinic compared with adults due to parental oversight.
On revenue timing, the CFO said Palynziq has continued to grow strongly even before the label expansion, citing growth of over 20% in each of the last two years despite being approved in 2017. However, he cautioned that revenue from new adolescent starts may lag because of the therapy’s gradual dosing induction and titration phase, which can take six to nine months or more before reaching steady-state efficacy doses, with lower doses generating less revenue early on.
When asked about competitive impacts to Palynziq, Cristin said the company is not seeing switching from Palynziq among patients who have reached maintenance dosing and are satisfied with therapy. She added that additional treatment options in the market could help bring “lost to follow-up” patients back into clinics, which BioMarin views as potentially expanding the treated population over time.
Finally, the CFO addressed quarterly variability, noting BioMarin has historically seen a Q4 ramp and a Q1 step down driven largely by timing of large international orders from national healthcare payers. He said the dynamic was “a bit exacerbated” entering Q1 2026, and also referenced modest U.S. inventory stocking—rising from about two weeks to three weeks on hand. Management said it expects 2026 quarterly trends to look similar to 2025, with Q1 as a low point, including Voxzogo being “on par with last year,” and reiterated that steady patient additions across products are the best indicator of underlying demand.
Looking beyond the Amicus transaction, the CFO said business development remains a “key part” of BioMarin’s strategy, but near-term priorities are closing and integrating Amicus and executing a deleveraging plan. He said the company is “levering up” for the acquisition, expects the deal to be dilutive this year due to interest expense, accretive in the first calendar 12 months after the acquisition, and “substantially accretive” beginning next year. BioMarin’s stated target is to reduce leverage to below 2.5x within two years after closing, while continuing to evaluate future clinical-stage pipeline transactions aimed at supporting long-term revenue growth.
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.
