BioMarin Stands Firm on 2027 Revenue Target Amid Ascendis Competition, Goldman Sachs Analyst Remains Bullish

Last week, Ascendis Pharma A/S ASND released topline data on Monday from a trial that included 84 children with achondroplasia, a genetic disorder that causes dwarfism and disproportionate short stature.

Goldman Sachs hosted an investor meeting with BioMarin Pharmaceutical Inc's BMRN Alexander Hardy (President and CEO) and Brian Mueller (EVP, CFO).

They reiterated financial guidance after reviewing their assumptions around Voxzogo competition post-Ascendis Pharma's better-than-expected Phase 3 achondroplasia data.

Also Read: Rare Disease-Focused BioMarin Narrows Sales Focus, Cuts Cost For Hemophilia A Gene Therapy; Lifts Annual Guidance.

The analyst notes that management believes a U.S./EU launch by Ascendis Pharma could violate its C-type natriuretic peptide (CNP) patents.

However, excluding this concern, the company still expects to reach approximately $4 billion in total revenue for fiscal year 2027, driven by growing Voxzogo adoption in the U.S., aided by its broad label without lower age restrictions, which could provide a competitive edge over Ascendis Pharma.

The company is targeting an adjusted operating margin of low-to-mid 40% (starting at 40% in 2026).

The new structure, announced at an investor event, covers three key business units: skeletal conditions, enzyme therapies, and Roctavian—its hemophilia A gene therapy.

BioMarin has been implementing a $500 million "cost transformation program" to contribute to non-GAAP operating margin targets set for 2026 and beyond.

Goldman Analyst writes, "We also see external business development as a potential source of upside and diversification beyond BioMarin's guidance. Nonetheless, we note continued investor debate on the outlook given increasing competitive pressure on BioMarin's key growth driver."

The analyst maintains a Buy rating with a price target of $139.

Price Action: BMRN stock is down 0.57% at $69.09 at the last check on Wednesday.

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