BioMarin Pharmaceutical's Kuvan Faces Competition from PTC Therapeutics' Sephience with Higher Pricing

BioMarin Pharmaceutical's competitor, PTC Therapeutics, has launched a drug priced at $490,000, significantly higher than BioMarin's Kuvan. Barclays analyst Gena Wang has updated the price target for PTC Therapeutics to $46, maintaining an Equal Weight rating. BioMarin's strong revenue growth, robust balance sheet, and positive business performance suggest potential undervaluation.

BioMarin Pharmaceuticals (NASDAQ: BMRN) has faced significant challenges in recent months, particularly with the potential approval of rival therapies for achondroplasia, a rare genetic condition. The company's share price has been impacted by the news that Danish Pharma Ascendis (ASND) has submitted a New Drug Application (NDA) for its therapy TransCon CNP, which is in direct competition with BioMarin's Voxzogo. Additionally, BridgeBio (BBIO) is developing infigratinib for the same indication, further intensifying the competitive landscape.

BioMarin's Q1 2025 earnings report revealed strong financial performance, with revenues increasing by nearly $100 million year-on-year, primarily driven by Voxzogo. The company's balance sheet remains robust, with $1.27 billion in cash and short-term investments and a long-term debt position of $(596m). For 2025, BioMarin has forecast an annual uplift in revenues of 10%, to $3.15 billion at the midpoint, and a forward price to sales ratio of ~3.7x.

However, the looming threat of competition from TransCon and infigratinib has raised concerns about BioMarin's ability to achieve its revenue targets. The company's Chief Commercial Officer has emphasized the broad label expansion and international footprint of Voxzogo, but the potential approval of TransCon by the FDA by November 30th this year poses a significant risk to BioMarin's market share.

PTC Therapeutics, a competitor, has launched a drug priced at $490,000, significantly higher than BioMarin's Kuvan. Barclays analyst Gena Wang has updated the price target for PTC Therapeutics to $46, maintaining an Equal Weight rating. This suggests that BioMarin's strong revenue growth, robust balance sheet, and positive business performance may be undervalued in the current market conditions.

In conclusion, while BioMarin Pharmaceuticals faces competitive threats from new entrants in the achondroplasia market, its strong financial performance and promising pipeline suggest potential undervaluation. Investors should closely monitor the FDA's decision on TransCon and BioMarin's ability to maintain its market share in the face of increased competition.

References:
[1] https://seekingalpha.com/article/4803723-biomarin-less-likely-m-and-a-target-as-rival-for-best-selling-drug-emerges