BioMarin Pharmaceutical Hits $1 Billion Revenue Milestone, Advances Rare-Disease Pipeline

Key Points

• BioMarin’s third-quarter revenue rose 32% to $1 billion, its first time reaching that milestone. Metabolic-disease revenue grew 44%, supported by the Amicus acquisition, including nearly 60% growth in POMBILITI and OPFOLDA sales.
• The company expects to capture about $220 million in annual operating-expense synergies from Amicus by 2028 and mostly complete integration by mid-next year, while targeting leverage below 2.5 times by then.
• BioMarin outlined upcoming pipeline milestones: BMN 440 data for hypophosphatasia are expected next year, BMN 333 phase 2 results in 2027, and BMN 820 phase 3 results in 2028. It is also advancing BMN 450 for Fabry disease, with no data-release date set.

BioMarin Pharmaceutical NASDAQ: BMRN is advancing a group of newly acquired rare-disease drug candidates while integrating Amicus Therapeutics and working to reduce debt, executives said during a company discussion.

Chief Financial Officer Brian Mueller said third-quarter revenue rose 32% to $1 billion, the first time the company reached that level. Revenue in the metabolic conditions business grew 44%, helped by the acquisition of Amicus and its Fabry disease treatment GALAFOLD and Pompe disease therapies POMBILITI and OPFOLDA.

Mueller said revenue from POMBILITI and OPFOLDA grew nearly 60%. BioMarin has identified roughly $220 million in non-GAAP operating expense synergies from the Amicus deal, which it expects to fully realize on an annual basis in 2028. The company expects the integration to be mostly complete by the middle of next year.

Hypophosphatasia candidate data expected next year

Chief Research and Development Officer Greg Friberg described BMN 440, an experimental oral treatment for hypophosphatasia, as a potential alternative to enzyme replacement therapies. The small molecule is designed to reduce production of inorganic pyrophosphate, which accumulates in patients with the condition and contributes to impaired bone mineralization.

Friberg said the company hopes the drug could reach muscle and joints as well as bone, potentially addressing pain and joint problems experienced by some patients. BioMarin plans to study the candidate in different pediatric and adult populations, but not in infants, whose disease involves other deficiencies that this approach would not address, he said.

The company expects to share data next year from an ongoing phase 1/2 study involving healthy volunteers and patients. The data will include measures of drug exposure and pyrophosphate levels. Friberg said BioMarin believes a 30% to 50% reduction in inorganic pyrophosphate would reach the range it is targeting.

Mueller said BioMarin estimates that approximately 9,000 people in the U.S. have a diagnosis of hypophosphatasia, but only 10% to 30% are receiving treatment. He said the wide range reflects limitations in the available information.

Fabry and growth-disorder programs

BioMarin’s recently acquired BMN 450 is an experimental Fabry disease enzyme replacement therapy designed for subcutaneous injection. Friberg said existing Fabry enzyme replacement therapies are administered intravenously.

Unlike GALAFOLD, which he said addresses an estimated 35% to 50% of patients with amenable mutations, BMN 450 is intended to work regardless of mutation. A phase 1/2 study is ongoing, and BioMarin has not committed to a date for publishing data. Friberg said the company is still developing its study plans and will need to work with regulators.

For BMN 333, BioMarin’s once-weekly candidate for achondroplasia, Friberg said phase 2 data are expected in 2027, with more precise timing to come. The study is testing three doses and includes a VOXZOGO control group. The company is investigating whether higher exposure can produce a greater effect on growth, rather than focusing on a non-inferiority approach, he said.

Friberg also said hypotension had not been observed in the phase 1 study. BioMarin will continue to monitor the benefit-risk profile, including whether faster growth could exacerbate other aspects of skeletal conditions.

Kidney program and VOXZOGO review

BMN 820, an oral candidate for the rare kidney condition focal segmental glomerulosclerosis, is in a fully enrolled phase 3 study with results expected in 2028, Friberg said. BioMarin holds U.S. rights to the partnered asset, which came through the Amicus acquisition. The study remains blinded and has passed a futility analysis.

Executives also discussed the potential expansion of VOXZOGO into hypochondroplasia. The regulatory decision date is May 30, 2027. Mueller said diagnosis is more complicated than for achondroplasia, but BioMarin’s experience and established treatment centers could support a launch if the indication is approved. The company has not decided whether to include a potential launch contribution in its 2027 guidance.

Mueller said BioMarin remains focused on acquiring clinical-stage assets while reducing leverage following the Amicus deal. Its target remains leverage below 2.5 times by the middle of next year, he said, without committing to a particular size for future transactions.

About BioMarin Pharmaceutical (NASDAQ:BMRN)

BioMarin Pharmaceutical is a biotechnology company focused on developing and commercializing medicines for people with rare genetic diseases. Its work includes research, clinical development, manufacturing and distribution of therapies for conditions that often have limited treatment options. The company's marketed treatments address disorders including phenylketonuria, mucopolysaccharidosis, late-infantile neuronal ceroid lipofuscinosis, achondroplasia and hemophilia A. Its products include Kuvan and Palynziq for phenylketonuria, Vimizim for Morquio A syndrome, Brineura for CLN2 disease, Voxzogo for achondroplasia and Roctavian for hemophilia A. Founded in 1997 and headquartered in San Rafael, California, BioMarin serves patients and healthcare providers in the United States and international markets, including Europe.

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