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Stock Watch: Advanced Therapies Deliver Fourth-Quarter Flop

Despite Obvious Clinical Benefits, Successful Commercialization Remains Elusive

 
• By Andy Smith

As drug pricing continues its ascent among hot button issues, the multi-million-dollar price tags for one-and-done advanced therapies, their justification and reimbursement all remain unaligned.

Stock Watch Image, Andy Smith
ANDY SMITH OFFERS A LIFE SCIENCE INVESTOR'S PERSPECTIVE ON BIOPHARMA BUSINESS

BioMarin Pharmaceutical Inc.’s fourth-quarter and full-year 2023 financial results announcement prompted a modest outperformance by its stock compared with the NASDAQ Biotech Index’s (NBI’s) 1% increase the day after BioMarin’s post-market announcement. Analysts and investors were focused on the majority of BioMarin’s commercial operations and sales of its advanced gene therapy product – Roctavian (valoctocogene roxaparvovec) for hemophilia A – seemed largely forgotten despite growing by nearly 240% on the third quarter of 2023. This was because the percentage of BioMarin’s fourth-quarter total sales contributed by Roctavian was so low that it looked more like a highly significant p-value.

Following Roctavian’s approval at the end of June 2023, BioMarin’s fourth-quarter commentary on it was limited to the “build-out of...

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