BioMarin Pharmaceutical Q2 Earnings Call Highlights

Key Points

• BioMarin’s second-quarter revenue nearly reached $1 billion, rising 20% year over year. The company raised its full-year revenue, VOXZOGO revenue and non-GAAP EPS outlook following strong rare-disease portfolio growth and the Amicus acquisition.
• Amicus therapies are expected to significantly expand BioMarin’s long-term growth. The company projects peak revenue of $1.4 billion for GALAFOLD and $1.2 billion for POMBILITI/OPFOLDA, with $220 million in annual cost synergies expected by 2028; the deal should become substantially earnings-accretive in 2027.
• VOXZOGO continued to grow despite new U.S. competition. Revenue increased 14%, global treated children rose more than 20%, and BioMarin said about 90% of U.S. patients remained on therapy while the drug stayed on track to become its first $1 billion product.

BioMarin Pharmaceutical NASDAQ: BMRN reported second-quarter 2026 revenue of nearly $1 billion, up 20% from a year earlier, as growth across its rare-disease portfolio and the recently completed Amicus acquisition supported results. The company raised its full-year revenue, VOXZOGO revenue and non-GAAP diluted earnings-per-share guidance, though it did not provide updated ranges during the call.

Chief Executive Officer Alexander Hardy said the quarter combined strong commercial execution with the close and integration of Amicus. BioMarin expects the acquired GALAFOLD and POMBILITI and OPFOLDA therapies to expand its growth profile through the mid-2030s, supported by international expansion, patient identification efforts and planned cost savings.

Amicus integration and growth outlook

BioMarin projected peak revenue of $1.4 billion for GALAFOLD, a treatment for Fabry disease, and $1.2 billion for POMBILITI and OPFOLDA, a combination therapy for Pompe disease. The company expects GALAFOLD revenue to grow at an approximately 10% compound annual rate from 2027 through 2032, while it expects POMBILITI and OPFOLDA to grow at a rate of at least 20% over the same period.

On a pro forma basis, GALAFOLD revenue increased about 10% year over year in the second quarter, while POMBILITI and OPFOLDA revenue rose more than 65%, according to Chief Commercial Officer Cristin Hubbard. GALAFOLD’s growth was driven by patient additions in established and newer markets, while POMBILITI and OPFOLDA added patients in the U.S. and recently launched geographies.

The company expects approximately $220 million in annual run-rate cost synergies to be fully realized in 2028, representing about a 50% reduction from Amicus’ 2025 non-GAAP operating expenses of $432 million. More than 70% of the expected savings are expected to come from general and administrative expenses, with the remaining savings primarily from research and development.

Chief Financial Officer Brian Mueller said the acquisition is still expected to be modestly dilutive in calendar 2026, though it is “close to breakeven.” BioMarin expects substantial earnings accretion to begin in 2027, with roughly half to slightly more than half of the planned synergies expected to be realized next year. The company also expects to reduce leverage about one year earlier than previously communicated.

Mueller said acquisition-related debt is expected to generate annualized interest expense of roughly $200 million, or about $50 million per quarter, based on current rates. That expense is included in non-GAAP results. Interest income is also expected to decline in the near term following the use of cash and investments to fund the acquisition.

VOXZOGO growth continues amid competition

VOXZOGO revenue grew 14% year over year in the second quarter, with double-digit growth in both the U.S. and international markets. The number of children receiving the treatment increased more than 20% globally from a year earlier, and approximately three-quarters of VOXZOGO revenue came from outside the U.S.

BioMarin said it raised its full-year VOXZOGO outlook following first-half performance and expectations for the remainder of 2026. Mueller said one international pricing negotiation closed with a favorable outcome, while another remained in process after initial setbacks. Growth in both the U.S. and global markets also contributed to the improved outlook.

The company is managing competition in the U.S. achondroplasia market following a competitor’s February launch. Hubbard said that approximately 90% of U.S. children using VOXZOGO had remained on treatment as of the end of July, based on the company’s available information. Hardy said BioMarin had observed approximately 10% of U.S. VOXZOGO patients switching, or fewer than 100 patients over roughly six months.

Hubbard said patients who switched were primarily seeking less frequent dosing or responding to injection fatigue. BioMarin said more than half of its new U.S. patient starts during the quarter were in children ages two and younger, an age group for which it said VOXZOGO remains the only approved treatment. Chief Research and Development Officer Greg Friberg estimated there are about 150 U.S. births annually involving infants with achondroplasia.

BioMarin expects both patient additions and ordering patterns to lift VOXZOGO revenue in the second half compared with the first half. The company said VOXZOGO is on track to become its first $1 billion product.

Pipeline and commercial updates

BioMarin submitted a supplemental new drug application for VOXZOGO in hypochondroplasia, following pivotal data reported during the quarter. Friberg said the company reduced the time from database lock to filing to 79 days through parallel work processes and technology-enabled efforts. Full Phase III results are scheduled for presentation at the ESPE meeting in September, and BioMarin plans to provide an update on the filing with third-quarter results.

The company estimates a global addressable hypochondroplasia population of approximately 14,000 patients. BioMarin said it is pursuing physician education, digital campaigns, genetic testing and other patient-identification initiatives ahead of a potential launch.

The Amicus transaction also added BMN 820, formerly DMX-200, to BioMarin’s pipeline. The oral CCR2 inhibitor is in Phase III development for focal segmental glomerulosclerosis, or FSGS. BioMarin holds exclusive U.S. commercialization rights, while partner Dimerix is responsible for operating the Phase III ACTION 3 study. The company expects Phase III data in 2028.

BioMarin’s Metabolic Conditions business, formerly called Enzyme Therapies, generated $695 million in second-quarter revenue, up 25% year over year including the acquired Amicus products. PALYNZIQ revenue increased 27%, aided by patient demand and U.S. ordering timing. The company also recently received European approval to expand PALYNZIQ’s label to adolescents ages 12 and older with PKU.

For the second half, Mueller said third-quarter revenue should be slightly above the second quarter, reflecting a full quarter of Amicus contributions. He expects the fourth quarter to be the company’s strongest of 2026, with ordering dynamics in select markets accounting for well over half of projected second-half revenue.

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.

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