BioMarin Crosses $1 Billion Quarterly Revenue, Targets 2028 Synergies

BioMarin Pharmaceutical booked $1 billion in third-quarter revenue, up 32% year over year — the first time the rare-disease biotech has crossed that mark in a single quarter. CFO Brian Mueller announced the milestone during a company discussion that also laid out integration economics, pipeline timelines and a deleveraging target of below 2.5 times leverage (net debt relative to EBITDA, a standard solvency gauge) by the middle of next year.

Where the growth came from

The metabolic conditions business drove the quarter, growing 44%. Mueller credited the Amicus Therapeutics acquisition, which brought in GALAFOLD for Fabry disease plus the Pompe disease therapies POMBILITI and OPFOLDA. Revenue from POMBILITI and OPFOLDA grew nearly 60%.

The deal is also producing cost savings. Mueller said BioMarin has identified roughly $220 million in non-GAAP operating expense synergies (cash cost reductions excluding accounting items), which it expects to fully realize on an annual basis in 2028. Integration should be mostly complete by mid-2026.

Takeaway: one acquisition delivered both the revenue record and a defined cost-savings number.

What's next for the hypophosphatasia pill?

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

He said the company hopes the drug could reach muscle and joints as well as bone, potentially addressing pain and joint problems in some patients. BioMarin plans to study the candidate in pediatric and adult populations — but not infants, whose disease involves other deficiencies this approach wouldn't address.

Data from an ongoing phase 1/2 study in healthy volunteers and patients arrive next year, including measures of drug exposure and pyrophosphate levels. Friberg said BioMarin believes a 30% to 50% reduction in inorganic pyrophosphate would hit its target range.

Mueller sized the opportunity: approximately 9,000 people in the U.S. have a hypophosphatasia diagnosis, but only 10% to 30% receive treatment — a wide range, he noted, reflecting limits in available data.

Takeaway: the market is largely untreated, and proof-of-concept data land within twelve months.

How do the Fabry and achondroplasia programs fit?

BioMarin also acquired BMN 450, an experimental Fabry enzyme replacement therapy designed for subcutaneous injection, versus the intravenous administration of existing therapies. Unlike GALAFOLD, which 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, but the company hasn't committed to a data publication date; Friberg said study plans are still developing and will require regulator engagement.

For BMN 333, a once-weekly achondroplasia (dwarfism) candidate, phase 2 data are expected in 2027. The study tests three doses against a VOXZOGO control group, investigating whether higher exposure produces greater growth effects rather than settling for non-inferiority. Friberg said hypotension wasn't observed in phase 1, and BioMarin will keep monitoring the benefit-risk profile, including whether faster growth could exacerbate other skeletal conditions.

What about the kidney trial and VOXZOGO expansion?

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

On VOXZOGO, executives discussed expansion into hypochondroplasia, with a regulatory decision date of May 30, 2027. Mueller said diagnosis is more complicated than for achondroplasia, but BioMarin's established treatment centers could support a launch. The company hasn't decided whether to include a potential launch contribution in 2027 guidance.

On capital allocation, Mueller said BioMarin remains focused on acquiring clinical-stage assets while reducing leverage post-Amicus — without committing to a size for future deals.

The message: record revenue, dated catalysts and a 2028 finish line for savings.