On a day when Vertex revealed trial data that indicate its next-generation cystic fibrosis (CF) treatment will soon be ready to grab the baton, the Boston company also presented (PDF) figures that show Trikafta remains formidable.
In the fourth quarter, Vertex's CF combo therapy Trikafta raked in $2.33 billion in sales, bringing its 2023 haul to $8.9 billion, a 16% increase from 2022. Trikafta appears poised to top the $10 billion mark this year, a significant milestone considering it was approved by the FDA in October 2019.
Helping Trikafta maintain its sales momentum has been its expansion into a younger population. In April of last year, the FDA signed off on the triple combo to treat children ages 2 through 5 who have particular mutations of CF. Two years before that, the U.S. regulator opened Trikafta to children ages 6 through 11.
“We continue to reach more patients, including younger ones, in core markets and select other countries,” Charles Wagner, Vertex’s chief financial officer, said during a conference call.
Vertex also sees growth in the CF market for the simple reason that patients are staying alive longer. The company has updated its estimate of the total CF patient population in North America, Europe and Australia from 88,000 to 92,000.
“We expect this trend to continue based on the real-world evidence we have generated on the clinical benefits of CFTR modulators. And this will also drive long-term growth,” chief operating officer Stuard Arbuckle said.
On Monday, the company reported that its investigational CF treatment, Vanza, achieved its main goals across several phase 3 programs, demonstrating non-inferiority against Trikafta for improving lung function and superiority to Trikafta in lowering levels of sweat chloride. The company plans to file for FDA approval in the middle of this year.
The biggest news for Vertex in the fourth quarter, of course, was it gaining FDA approval for sickle cell disease treatment Casgevy. Then last month, the regulator tacked on a nod for the gene-editing therapy to treat another debilitating blood disorder, beta thalassemia.
Vertex said that it has established 12 authorized treatment centers (ATCs) in the U.S., three in Europe and one in Saudi Arabia. As it launches Casgevy, the company said it will provide quarterly updates on its ATC numbers and how many patients are in the cell collection phase. The first commercial patients will begin treatment in the “coming weeks,” Arbuckle said.
Overall, Vertex reported revenue of $9.9 billion for 2023, which was an 11% increase from 2022, making it the ninth straight year the company has accomplished revenue growth at double-digit percentages, Wagner noted.
In 2024, Vertex is guiding to sales of between $10.55 billion and $10.75 billion. While that estimate includes some contributions from the launch of Casgevy, the company stressed that this is a foundational year “as we ramp up patient initiations and build toward a multi-billion dollar market opportunity over time,” Wagner said.