Cystic fibrosis (CF) specialist Vertex is tracking two newer launches that speak to its efforts to expand its prowess beyond those roots. Despite mixed early results for those rollouts so far, the company is forecasting a major acceleration of non-CF revenues this year.
Vertex's efforts to diversify its portfolio beyond its leading CF treatments have so far manifested in gene therapy Casgevy, which was approved in 2023 for sickle cell disease and in 2024 for beta thalassemia, and non-opioid pain med Journavx.
The latter hit the market at the start of last year and has so far collected $59.6 million in sales. When combined with $116 million in Casgevy sales from last year, the company's non-CF meds generated $175.6 million in 2025.
But the company figures the non-CF business can quickly gain pace. This year, Vertex aims to take in $500 million or more from products outside its CF franchise, fitting into its $12.95 billion to $13.1 billion overall guidance.
Journavx is coming off a foundational year, Vertex executives explained in presenting the company's financial results. In 2025, the company worked to build a broad prescriber base for the drug across multiple specialists and expand coverage across major payers and hospital access pathways.
These efforts are meant to "create a strong long-term foundation for years of growth," Chief Commercial Officer Duncan McKechnie explained on Vertex's earnings call Thursday.
So far, the pain med has seen "positive reception" from patients with surgical and non-surgical pain, Vertex said in its earnings presentation (PDF). Still, the drug's $26.7 million in fourth-quarter sales missed analysts' consensus of $32.9 million, Evercore ISI analysts pointed out in a note to clients.
"Perhaps most importantly, we estimate that about 420,000 Americans benefited from the inclusion of [Journavx] in their treatment journey as an effective, well-tolerated non-opioid option for moderate to severe acute pain," McKechnie said.
The goal in 2026 is to increase prescriptions threefold from the roughly 550,000 it achieved in 2025.
As it stands, the prescriptions are about a 50/50 split between hospital and retail channels, McKechnie said. To drive further prescriptions, Vertex is piloting a TV ad campaign in "select markets," and the company is also looking to double the size of its field force this year, the executive added.
Casgevy's blockbuster potential
Meanwhile, the company continues to push toward realizing Casgevy's "multi-billion dollar potential," McKechnie said. With $116 million in full-year sales, however, it seems the gene therapy still has a long ways to go on the road to blockbusterland.
The executive pointed to the drug's "excellent" fourth quarter, which brought in $54 million in revenue as 11 new patients initiated the treatment process and 30 received infusions. This outcome beat Evercore ISI's projection of $41.1 million, according to the analysts.
However, upcoming earnings periods may see quarter-to-quarter "variability" in infusion rates given the duration of the patient journey and the impact of patients' choices with regard to infusion timing.
"We expect this to smooth out in 2027 and beyond as the number of patients at all stages of the treatment journey continues to build," McKechnie maintained.
Although it has broadened its reach outside of its flagship cystic fibrosis franchise, Vertex continues to gain steam in its mainstay indication. Its latest innovation, Alyftrek, launched in late 2024 as a once-daily triple combination for CF patients with certain mutations.
The drug collected $838 million in its first year on the market, but Vertex sees a chance to broaden its reach to younger patients after recently wrapping an Alyftrek study in children aged 2 to 5. The results were "unprecedented" for the age group, Vertex said, leading the company to prepare to submit its bid for a label expansion in the first half of 2026.
As it stands, Vertex's clutch of CF medicines can treat nearly 95% of all people living with the disease in core markets, including those as young as one month old. The majority of the company's revenue comes from its 2019-approved Trikafta, which is sold as Kaftrio in Europe. In 2025, the CF med generated sales of $10.3 billion, helping grow Vertex's total yearly revenue to $12 billion.
Trikafta remained "surprisingly resilient" in 2025, but William Blair analysts expect switches to newer Alyftrek to "modestly erode" its lead in 2026, the analysts wrote in a note to clients.