Vertex Pharma Gets FDA Approval for Combo CF Drug, Sets $292,000 Price

Xconomy Boston — 

[Updated, 2/13/18, see below] The FDA has approved a new Vertex Pharmaceuticals cystic fibrosis drug, clearing the way for the Boston company to bring to the market its third treatment for the inherited disorder.

The new Vertex (NASDAQ: VRTX) drug pairs the already approved ivacaftor (Kalydeco) with the newly approved drug tezacaftor. Vertex set a list price for its ivacaftor/tezacaftor (Symdeko) combination at $292,000 per year.

Cystic fibrosis (CF) is a genetic disorder that causes a thick buildup of mucus in the lungs and pancreas. The approval of ivacaftor/tezacaftor strengthens Vertex’s case that drug combinations are the right approach to treating the disease. The company’s first FDA-approved CF drug, ivacaftor, treats a small subset of those who have a certain genetic mutation—roughly 5 percent of CF patients. Combining ivacaftor with other drugs, Vertex reasons, makes treatment available to more patients.

Vertex’s second FDA approved CF treatment was an ivacaftor/lumacaftor (Orkambi) combination. With approval of the ivacaftor/tezacaftor combo now in hand, Vertex estimates its product line could treat up to 40 percent of the 70,000 CF patients worldwide.

[Updated with analyst comments] Will the new medicine fare better than Orkambi? That combination treats many of the same CF groups but has limitations—like side effects such as chest tightness—that have limited its uptake and caused a portion of patients to discontinue treatment. Orkambi is used by less than 60 percent of the possible addressable CF population because of these problems, wrote Leerink analyst Geoffrey Porges in a research note Tuesday morning.

Vertex has touted the tezacaftor combination as a possible alternative for people who have trouble with or aren’t eligible for Orkambi or ivacaftor alone. When Vertex revealed its Phase 3 data last year, several CF experts told Xconomy that more time was required to show if the ivacaftor/tezacaftor combination is superior to Orkambi. Vertex is betting it is, having priced the medicine roughly 7 percent higher than Orkambi’s $273,000-per-year cost, before discounts.

Porges wrote that FDA label shows the new combination is better than Orkambi, justifying a higher price. The drug combo is more effective, doesn’t have monitoring requirements for blood pressure, and doesn’t include warnings for respiratory problems, meaning patients are “more likely to try, and continue on Symdeko,” he wrote.

In the U.S., “we do not expect much resistance to this premium from payers, physicians or patients, given the considerable advantages of Symdeko over Orkambi,” he added. Porges expects more problems in Europe, where Vertex has had a much tougher time getting reimbursement for Orkambi. Vertex expects European regulators to issue a decision for ivacaftor/tezacaftor in the second half of this year.

Beyond its two-drug combinations, Vertex also has results from early and mid-stage studies from three-drug combinations that suggest it could treat up to 90 percent of CF patients.

Here’s more about Vertex and its strategy of using drug combinations to treat CF.

Ben Fidler contributed to this report.