Dive Brief:

• In a highly expected move, the Food and Drug Administration on Monday approved Vertex Pharmaceuticals Inc.'s latest combination treatment, Symdeko, paving a way for the biotech to serve a greater portion of the cystic fibrosis patient population.
• Vertex set Symdeko's annual list price at $292,000, sandwiching it between the $272,000 and $311,000 it respectively costs for a year's supply of the company's other marketed CF products, Orkambi and Kalydeco.
• Largely referred to as tez/iva, Symdeko pairs a new corrector, tezacaftor, with the active ingredient in Kalydeco, ivacaftor. Assuming the combo would gain approval and Orkambi uptake would continue to grow, Vertex executives estimated about 44,000 CF patients — more than half the global population — would be eligible to take the company's treatments.

Dive Insight:

The FDA's decision not only gives Vertex a third ready-for-market CF drug, but also makes it easier for the biotech to conduct clinical investigations for triple combos.

Last month, Vertex revealed plans to take two triplets into late-stage testing: one consisting of the tez/iva backbone and a next-generation corrector, VX-659, the other consisting of tezacaftor, an investigational potentiator, VX-561, and another next-generation corrector, VX-445. Should the triplets gain approval, Vertex believes its portfolio could treat up to 90% of the CF population.

But to stand a chance at approval, the triple combos will need to show they're more effective than the two-drug combos in head-to-head trials, according to Jefferies analyst Michael Yee. An FDA OK for tez/iva provides a straightforward way of doing just that, Yee noted in November, allowing Vertex to use the product as a control group so it can demonstrate the added benefit of pairing VX-659 or VX-445 to already effective drugs.

Such an advantage puts the company in an even stronger position against fellow CF drugmakers like Galapagos NV.

In a Feb. 12 note, Jefferies called Vertex the "cleanest growth story in biotech," and predicted the company's revenues and earnings could triple in the next three to five years. Vertex recorded $2.49 billion worth of revenue last year, reflecting an almost 50% increase from 2016.

Symdeko will surely lift bottom lines in 2018. Though the drug is pricier than Orkambi, it has demonstrated stronger efficacy and safety in the clinic. Yee predicted that patients who stopped taking Orkambi in the last couple years could find added benefit by starting Symdeko treatment — setting Vertex up to fetch about $100 million to $200 million from the new drug this year.

"Symdeko’s price reflects its demonstrated clinical benefits in CF, including a favorable safety/tolerability profile, and our ongoing commitment to reinvesting in the development of new medicines for the more than half of people with CF who still do not have a medicine to treat the underlying cause of their disease," Vertex told BioPharma Dive in an email.

"Our first two medicines, Kalydeco and Orkambi, are widely covered by public and private insurers in the U.S. and we expect the case will be the same for Symdeko," the company added.

Vertex shares were down less than 1% to $151.40 apiece at market's open Tuesday, but climbed to $153.85 apiece by mid-morning.