Vertex

With new approvals on the horizon and the closely watched Casgevy (Exagamglogene autotemcel) launch picking up steam in sickle cell disease and beta thalassemia, “there has never been a more exciting time to be at Vertex,” chief operating officer Stuart Arbuckle said on the company’s third-quarter earnings conference call.

With the first Casgevy revenues coming in and the company's established cystic fibrosis portfolio firing on all cylinders, Vertex brought in a quarterly haul of $2.77 billion, a 12% increase from the same period last year. The number beat the market consensus of $2.66 billion and William Blair’s estimate of $2.64 billion, the firm's analysts wrote in a note to clients.

The performance led Vertex to raise its full-year product revenue guidance to a range of $10.8 billion to $10.9 billion. Previously, Vertex had expected to generate $10.65 billion to $10.85 billion in sales this year.

CRISPR Therapeutics-partnered Casgevy’s official entrance to the commercial market is a crucial milestone following months of efforts to lay the groundwork for the launch. So far, Vertex has set up 45 authorized treatment centers for the gene-editing therapy and is “well on our way” to meeting its goal of 75 centers globally, Arbuckle said. Forty patients have already had at least one cell collection, doubling last quarter’s 20 patients.

The $2.2 million therapy has come a long way since its groundbreaking sickle cell disease FDA approval in December and subsequent January nod in beta thalassemia. As Vertex looks to tap into the Middle Eastern market of around 23,000 patients—on top of its estimated patient pool of 35,000 in the U.S. and Europe—the drugmaker remains “confident in our view that Casgevy represents a multibillion-dollar opportunity,” Arbuckle said.

“We have built a strong foundation for this transformational therapy, and we look forward to the momentum it will carry into 2025," he added.

At the same time, Vertex is keeping an eye on increasing manufacturing capacity. In September, the company inked a long-term supply agreement with CDMO giant Lonza, which will help produce the therapy from its manufacturing digs in the Netherlands.

Next up for Vertex is a January FDA action date for its acute pain med suzetrigine. With field teams hired and trained, the company is "launch-ready" as it looks to bring a new option to market that could benefit the 80 million patients in the U.S. who seek a pain prescription each year, Arbuckle said.

January will also see an FDA decision on Vertex’ newest triple combo cystic fibrosis medicine, vanzacaftor triple. The drug is expected to reach the more than 5,000 patients who don’t make any cystic fibrosis transmembrane conductance regulator (CFTR) protein, adding to the more than 65,000 people treated with the company's existing cystic fibrosis brands as of July.