BioMarin Targets Amicus Synergies as VOXZOGO Nears $1B Milestone

BioMarin expects $200 million in annual non-GAAP cost savings, with most synergies realized in 2027 and full realization in 2028. The deal is expected to accelerate deleveraging, with leverage falling below 2.5 times by mid-2027. The drug delivered 20% quarterly patient growth despite U.S. competition, and BioMarin expects revenue to reach approximately $1 billion this year. Potential approval in hypochondroplasia could provide additional upside. As cash flow improves and leverage declines, the company expects to regain capacity for deals of up to $5 billion, potentially rising to $6 billion by 2028, while advancing programs including ALE1 and BMN 333.

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.

BioMarin expects peak sales of approximately $1.4 billion for GALAFOLD and $1.2 billion for POMBILITI + OPFOLDA by the mid-2030s. Hardy said the opportunity for GALAFOLD is centered on improving Fabry disease diagnosis and treatment rates, supplemented by geographic expansion. He said the company believes it can more than double the number of U.S. patients on GALAFOLD by the mid-2030s. 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 tr...