The biotech beat Wall Street’s third-quarter revenue forecast by 6%, driven by increased uptake of its achondroplasia drug Voxzogo. However, William Blair downgraded BioMarin’s shares to market perform due to a “lack of near-term catalysts” and uncertainty around Voxzogo’s potential revenue growth.
BioMarin Pharmaceuticals on Tuesday announced $746 million in total revenue in the third quarter, 28% year-over-year growth driven by the continued strong uptake of its therapies for rare genetic diseases.
The biotech beat consensus estimates, surpassing Wall Street’s expected Q3 revenue of $703 million by 6%. BMO Capital Markets analyst Kostas Biliouris in an investor note attributed BioMarin’s strong performance to the continued growth of its achondroplasia medicine Voxzogo, with sales of $190 million jumping 54%. There are now over 3,800 patients on Voxzogo, according to BioMarin, up from around 3,500 in the second quarter.
Still, Wall Street anticipated Voxzogo generating $195 million for BioMarin in Q3, a bar the company missed by 3%.
Roctavian, a gene therapy approved in June 2023 for hemophilia A, brought in $7 million in the quarter, in line with the analyst consensus. And while its relative contribution to BioMarin’s total Q3 revenue is low, Roctavian presents a high-growth opportunity for the biotech with 600% sales growth versus the same period the year prior.
Biliouris said that this “continued uptake” of Roctavian reflects the company’s efforts to focus on the U.S., Italy and Germany markets, potentially paving the way for hitting around $60 million in revenue for the gene therapy by 2025.
Truist Securities analyst Joon Lee also credited BioMarin’s Sanofi-partnered enzyme replacement therapy Aldurazyme for its Q3 beat. Sales of Aldurazyme, indicated for mucopolysaccharidosis I, jumped 407% in the quarter bringing in $71 million and beating the consensus expectation of $30 million by $137%.
Encouraged by its strong Q3 showing—and by the continued growth of its products—BioMarin on Tuesday raised its full-year 2024 revenue guidance to between $2.79 billion and $2.825 billion. The biotech previously expected a range of $2.75 billion to $2.825 billion this year.
Despite BioMarin’s promising outlook for 2024, Lee warned about impending competitive pressure on Voxzogo. Last month, Ascendis Pharma revealed strong pivotal data for its investigational drug TransCon CNP, also known as navepegritide, which showed significant improvements in annual growth velocity in children with achondroplasia. Ascendis is gearing up for a regulatory filing for TransCon CNP in the first quarter of 2025.
“In light of better-than-expected data, we’re left to wonder what further impact competition from TransCon-CNP may have on BioMarin’s mid- to long-term guidance,” Lee said.
Likewise, William Blair analyst Sami Corwin in a note to investors said that despite the Q3 beat, her firm downgraded shares of BioMarin to market perform. “We do not think its clinical pipeline will be a source of significant value creation in the near term,” according to Corwin. “Voxzogo remains the key top-line growth driver for the company, but the threat of competition could erode its revenue growth and market share.”
