FDA nod for Vertex's improved CF combo, Symdeko, opens $200M door this year

Vertex CEO Jeffrey Leiden
Vertex executives, led by CEO Jeffrey Leiden, predict their cystic fibrosis meds can generate $2.65 billion to $2.8 billion in sales this year. (Vertex)

Step aside, Orkambi. Vertex’s new-and-improved cystic fibrosis combo is here.

On Monday, the FDA approved Symdeko, a combination of new compound tezacaftor and ivacaftor, a drug already on the market solo as Kalydeco. Last year, the pair showed it could outdo Orkambi—which marries ivacaftor and lumacaftor—in improving lung function for patients with specific mutations, and its new label bears some major advantages over its predecessor’s, too.

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For one, Symdeko’s label lacks monitoring requirements for blood pressure and for low lung function patients, Leerink Partners analyst Geoffrey Porges wrote in a note to clients. It also lacks Orkambi’s warning on respiratory events.

Those “negative attributes” of Orkambi had kept market penetration to below 60% of eligible patients, Porges noted. But “with the label advantages and double the efficacy, Symdeko could ... provide robust top-line growth for Vertex.”

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Just how much top-line growth are we talking? Porges expects Symdeko to “ultimately to produce as much as 30-40% more revenue in a given market than Orkambi,” and he predicts the new med can help Vertex’ CF products rake in $2.84 billion this year. Jefferies’ Michael Yee, meanwhile, wrote to his own clients that “we do think Symdeko could contribute $100-200M+ this year based on identified patients who came off Orkambi in the last two years.”

“We think revenue and earnings can triple over the next three to five years,” he added. 

Vertex's own revenue forecasts in cystic fibrosis, for their part, sit at $2.65 billion to $2.8 billion for the year. Analyst consensus sits between Vertex's estimates at $2.75 billion, though Symdeko’s wholesale price—$292,000, or 7% higher than Orkambi’s—“may lead to a small boost in estimates,” Porges pointed out.

Importantly, the new approval bodes very well for Vertex’s pipeline, too—specifically, a closely watched three-drug cocktail that will include both tezacaftor and ivacaftor.

It “further de-risks the ‘triple pill,’” Yee wrote.