BioMarin Pharmaceutical (NASDAQ:BMRN) said it is progressing with the integration of Amicus Therapeutics while pursuing additional business-development opportunities, as executives outlined growth plans for newly acquired rare-disease products, VOXZOGO and several pipeline programs at Morgan Stanley’s Global Healthcare Conference.
President and CEO Alexander Hardy said the company has delivered business growth, margin expansion and efficiency improvements while integrating Amicus. He described the transaction as primarily driven by the opportunity to accelerate revenue from GALAFOLD and POMBILITI + OPFOLDA, as well as realize operating-cost synergies. Diversification of BioMarin’s growth profile was a secondary benefit, he said.
Amicus Integration and Financial Targets
For POMBILITI + OPFOLDA, Hardy said the commercial opportunity is more dependent on patients switching from other Pompe disease therapies as evidence and real-world data develop. He characterized that market as a longer and slower opportunity than GALAFOLD’s.
BioMarin expects $200 million in non-GAAP expense savings from the Amicus transaction. Hardy said Amicus had about 505 employees and BioMarin expects to retain approximately 192 over the long term. The company primarily retained commercial and medical-affairs personnel supporting GALAFOLD and POMBILITI + OPFOLDA, while about 70% of the savings are expected to come from general and administrative expenses, with the remainder from duplicated research and development functions.
Executive Vice President and CFO Brian Mueller said the integration planning has moved beyond high-level targets, with employee decisions communicated and operational cutover plans established across functions. He said integration will continue through this year and into next year because some systems and processes are qualified and regulated. BioMarin expects to realize most synergies in 2027, with 2028 representing the first full year of full synergy realization.
Mueller said BioMarin expects the transaction to be accretive in its first year, with substantial accretion beginning next year. The additional cash flow has allowed the company to advance its deleveraging target. BioMarin now expects leverage to fall below 2.5 times by the middle of 2027, nearly a year earlier than its initial target of within two years of the transaction’s announcement.
Hardy said reaching that target would restore the company’s pre-Amicus acquisition capacity for deals of up to $5 billion. Mueller said that, with continued EBITDA growth, capacity could reach approximately $6 billion by 2028. BioMarin recently announced and closed a deal to add Alesta Therapeutics’ ALE1 program to its pipeline, and Hardy said the company intends to continue pursuing transactions while it deleverages.
VOXZOGO Growth and Competitive Landscape
Hardy said VOXZOGO continued to grow despite the entry of a competitor in the U.S. market. He cited 20% quarterly patient growth and said the company raised its full-year outlook. BioMarin expects VOXZOGO to reach $1 billion in revenue this year, which would make it the company’s first blockbuster product, according to Hardy.
He said most new VOXZOGO patient starts have been in the zero-to-two-year-old population, where BioMarin has an indication and data advantage that will remain exclusive for several years. The company is also awaiting a potential FDA approval for VOXZOGO in hypochondroplasia, an opportunity not incorporated into its 2026 outlook.
BioMarin has reached a settlement with Ascendis that provides BioMarin a 20% royalty rate in the U.S. and an 18% royalty rate in Europe, Brazil and South Korea. Hardy said the arrangement allows BioMarin to benefit if Ascendis’ TransCon treatment brings additional patients into the market.
Hardy added that BioMarin markets VOXZOGO in 55 countries and has an 80-country commercial footprint. He said market access outside the U.S. is more complex and requires local expertise, giving BioMarin an advantage over newer entrants.
Established Products and Pipeline
BioMarin said PALYNZIQ grew 27% to $135 million, aided by the U.S. adolescent-label launch. Hardy estimated the 12-to-17-year-old population could add roughly 10% to the addressable patient opportunity. He said the therapy’s efficacy may appeal to families preparing adolescents for greater independence, including college.
Hardy also said VIMIZIM has the potential to exceed $1 billion in revenue. He noted that quarterly results can be affected by international ordering patterns and government purchases, but said VIMIZIM grew 7% in 2025 and has historically grown at around that pace. Mueller encouraged investors to focus on full-year results and guidance because of quarterly timing effects.
On pipeline development, Hardy said BioMarin’s BMN 333 showed more than 13-fold higher free CNP exposure than another long-acting CNP agent in a Phase I healthy-volunteer study. The company is conducting an operationally seamless Phase II/III study to determine whether higher free CNP levels translate into improved annualized growth velocity and other health benefits. BioMarin expects to have Phase II findings around the middle of the year.
Regarding ALE1, Hardy said the oral candidate for hypophosphatasia could potentially address bone manifestations as well as symptoms such as muscle pain and fatigue by reducing pyrophosphate at its source. He said approximately 6,000 U.S. patients are diagnosed with the disease, but only an estimated 10% to 30% receive treatment.
About BioMarin Pharmaceutical (NASDAQ:BMRN)
BioMarin Pharmaceutical Inc is a biotechnology company focused on developing and commercializing therapies for rare and serious diseases. Founded in 1997 and headquartered in San Rafael, California, the company specializes in treatments for conditions that often have limited or no available therapeutic options, including inherited metabolic disorders, skeletal diseases, and certain bleeding disorders.
BioMarin’s marketed products include Vimizim for mucopolysaccharidosis type IV A, Naglazyme for mucopolysaccharidosis type VI, Brineura for late-infantile neuronal ceroid lipofuscinosis type 2, Palynziq and Kuvan for phenylketonuria, and Voxzogo for achondroplasia in children with open growth plates.
