New Products

On a roll, Vertex grabs FDA OK for CF double shot Symdeko — prices at $292,000

Close on the heels of its impressive data on new triple combos for cystic fibrosis, Vertex reported out today that the FDA has approved its double using tezacaftor/ivacaftor.

In a drive to continually expand on its revenue, Vertex announced that it will price the combo at $292,000 a year, putting it in line for steadily swelling revenue numbers for the year ahead.

Jeffrey Leiden

The combo will be marketed as Symdeko to anyone 12 and over with at least one mutation that is responsive to the double.

In anticipation of the approval, Leerink recently boosted its estimated revenue at Vertex to $2.84 billion this year — up from $2.17 billion in 2017. And they are betting Vertex hits a cool $3.75 billion in 2019.

Jefferies’ Michael Yee put it this way:

More notably VRTX announced: (1) gross pricing of $292k which is slightly lower than Kalydeco gross of $311k but above Orkambi’s gross of $259k….so this means pts swapping from Orkambi to Smydeko would have a slightly accretive benefit (modest given we don’t see that much ‘swapping’) but because of the added efficacy and less side effects (chest tightness) the improved drug does have a slightly higher gross price so pts who have an option and came off Orkambi in the last 1-2 years can now benefit from Symdeko and this is all accretive as they’re not getting revenues from those pts (we est $100-500M benefit in 2018-2020). (2) 2018 CF guidance of $2.6-2.8B – this is right in-line w/ our and consensus $2.7B….we do think Symdeko could contribute $100-200M+ this year based on identified pts who came off Orkambi in the last 2 years (see our VRTX-CF-APP model).

Looking to hedge its bets on a big gamble that it can greatly expand the number of CF patients it can help, Vertex is taking two triples into pivotal trials. The 445 triple actually just barely edged out 659 for the high-dose response, with a 13.8% improvement in FEV1. But the second highest dose only came in at a single-digit improvement rate, with a lower rate of reduction for sweat chloride as well as two troubling discontinuations in one of the drug arms.

“Today is an exciting day for the CF community. The approval of SYMDEKO, our third disease-modifying CF medicine, offers many patients an important new treatment option,” said Jeffrey Leiden, Vertex’s chairman, president and chief executive officer. “This approval is an important milestone in our journey to treat every person with CF, and we remain committed to urgently advancing our efforts to develop new medicines that treat the underlying cause of CF for the many people still waiting.”


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