The triple combo era in cystic fibrosis is here thanks to a Monday Vertex Pharmaceuticals approval—and it’s here earlier than industry watchers expected. A lot earlier.
Five months ahead of schedule—and just three months after the company submitted its regulatory application—the FDA greenlighted Trikafta to treat CF patients over the age of 12 with either one or two copies of the delF508 CFTR gene mutation.
The drug will “be available at pharmacies in a few weeks,” Jefferies analyst Michael Yee wrote in a note to clients, and it’ll bear a list price of $311,000—slightly above what Wall Street had predicted, but in line with the price of Kalydeco, the company’s first cystic fibrosis drug and one of the three that make up Trikafta.
Yee sees Trikafta zooming to blockbuster territory as early as next year, and SVB Leerink's Geoffrey Porges estimates 2025 sales north of $6 billion. But some of that explosive growth is likely to come at the expense of Vertex's older meds, thanks to the new drug's "whole new level" of efficacy, Porges said.
It’s no surprise Trikafta’s review was a speedy one, considering it “benefited from a full deck of FDA accelerated development programs and pathways” such as priority review, and fast track and breakthrough therapy designations, Porges wrote in a note to clients.
Still, the timing appears to have surprised even Vertex, Porges said, pointing to the company’s “lack of an accompanying company quote, press release or conference call announcement.”
If the FDA did catch it off guard, though, Vertex isn’t complaining. The go-ahead will help the company reach 90% of cystic fibrosis patients in the U.S., compared with the 50% that its drugs are currently cleared to treat.
As a result, Porges has predicted Trikafta could hit $4.6 billion in sales by 2023 and $6.6 billion in 2025, though “this timing suggests to us that the revenue ramp could occur earlier, and might therefore be steeper, than we had previously forecast,” he wrote.
Jefferies’ Yee, for his part, foresees Trikafta breaking the blockbuster barrier next year. “We predict a big launch will now happen even earlier, putting the stock ‘in motion’ for beating estimates earlier than Street was preparing for,” he added.
Meanwhile, the launch of Trikafta could spell the end for Vertex’s older, two-drug therapies. “Trikafta takes the efficacy benefit for CF patients to a whole new level compared to Symdeko and Orkambi, which are likely to fade away relatively quickly in all but the most stable patients (or those unable to access the triple due to geography or age),” Porges wrote.
And on the topic of geography, the way Porges sees it, the Trikafta approval “is likely to increase the pressure on resistant payers such as England” to strike a deal with the company, he said.