In April, BioMarin forecast that its new gene therapy Roctavian would bring in $50 million to $100 million in sales by the end of 2023. Instead, it’s collected just $3.5 million in revenue for a mere three patients.

While the company had significantly lowered expectations late last year, and Wall Street had lowered them further, the paltry sales are yet another reminder of just how challenging it can be to deliver complex, high-cost new gene therapies to the market.

On an investor call Thursday, BioMarin CEO Alexander Hardy said the slow uptake isn’t a surprise for a “pioneering new therapy.”

“We’re still very much at the early stage with Roctavian,” Hardy said, according to a transcript from AlphaSense. “We are seeing progress in terms of market access, market activation. So, we wanted to see that run through 2024. We also think probably into 2025 is when we’ll be able to really determine. In the meantime, we’re spending wisely and cautiously supporting the launch.”

BioMarin said in its Q4 press release that the commercial team is focusing on increasing uptake of Roctavian, including site readiness, educating doctors and patients, and identifying people eligible for treatment.

In November, BioMarin announced a three-year deal with the German National Association of Statutory Health Insurance Funds for reimbursement of its gene therapy. Two of the three patients that have been treated so far were in Germany and one was in the US. BioMarin’s list price for Roctavian in the US is $2.9 million, which translates to a net price of about $1.9 million after typical discounts and rebates, according to the company.

But Leerink Partners analysts wrote Friday that they are lowering their pricing estimates for Roctavian in the EU to $900,000 per patient, down from $1.45 million, and decreasing peak sales estimates to about $1 billion from $2.3 billion “given the lack of visibility on the launch.”

Hardy joined as BioMarin’s CEO in November, the same month that activist investor Elliott Investment Management bought over a $1 billion stake in the biotech. On Thursday’s post-earnings call, the company faced questions about whether it might consider divesting from the gene therapy business or Roctavian altogether.

The company has said that it’s begun a review of all its R&D programs to decide which would move forward, and will share the update at its investor day later this year.

“As part of that, we’ll assess: Do we have the right exposure to gene therapies in our pipeline, is it something we want to double down on, stay the course or take a lower exposure to gene therapies?” Hardy said.

Check out our AAV CDMO service to expedite your gene therapy research
About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Scientists develop potential stealth cancer therapy

Credit: ACS Central Science (2024). DOI: 10.1021/acscentsci.4c00559Sneaking by cancer's defenses, by disguising tumor-fighting antibodies inside the molecules cancer uses to nourish tumor growth, is the basis of a novel therapy from Yale Cancer Center researchers at...

read more

Related Services

Plasmids GMP Services

Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.

AAV GMP Services

Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
aav icon

Technology Platforms

PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.