Vertex cancels plans for CF triple combo testing in France, citing failed Orkambi negotiations

After failing to reach a reimbursement agreement with French officials on its cystic fibrosis (CF) drug Orkambi, Vertex has cancelled plans to test an investigational triple combination at several sites in the country. 

Patient advocates say it appears to be a form of "blackmail," but the drugmaker argues it can't enroll patients when there's no guarantee they'll have long-term access to innovative treatments. 

In a statement on the situation this week, patient groups Overcoming Mucoviscidosis, the Grégory Lemarchal Association and the French Cystic Fibrosis Society said trials for a new triple therapy designed to treat the largest number of CF patients to date were "highly anticipated" by the community. But reimbursement negotiations for Orkambi haven't gone anywhere, and so the drugmaker has pulled plans to test an investigational therapy there. 

"This situation, which sounds like a condemnation for those involved in the fight against cystic fibrosis in France, is unacceptable for both patients and carers, and appears as a form of blackmail," the statement said, according to a translation. 

Vertex's director of external communications Heather Nichols said via email that the company is setting out to cure CF and has made "significant advances by discovering and developing the first medicines to treat the underlying cause of this disease." 

She added that the drugmaker wants all patients to be able to access its current and future medication. But, even though the company negotiated for 18 months and made six different offers, "we haven’t been able to reach a reimbursement agreement in France for Orkambi." 

"The French counteroffer was an 80% discount from our proposal and valued Orkambi similarly to a 20-year old medicine that only treats symptoms of the disease," she wrote. "If this is an indication of how France views innovative medicines, we cannot, in good faith, enroll patients in clinical trials for medicines they may not have access to in the future." 

The drugmaker said its offer is still on the table and that it urges France "to join countries like the U.S., Austria, Ireland, Germany and many others to provide patients sustainable access" to its drug. Similarly, the patient groups urged Vertex to reconsider its cancellations and to continue negotiations.

It's not the first time the company has failed to strike a reimbursement deal in Europe. Back in 2016, England's influential cost watchdogs at NICE gave Orkambi a thumbs down, saying its £104,000 price tag was too high to be a “cost-effective use of NHS resources.” Those negotiations are ongoing.

Orkambi costs $273,000 per year before discounts in the U.S., according to a recent note from Leerink analyst Geoffrey Porges.

RELATED: NICE sticks to its guns and turns down Vertex's Orkambi even as patients plead

A combination of lumacaftor and ivacaftor, Orkambi won EU approval back in November 2015.

The drugmaker recently announced plans to move investigational candidate VX-659 into phase 3 testing in combination with new compound tezacaftor and ivacaftor, marketed as Kalydeco. Beyond that, it's also working to push VX-445 into late-stage testing with tezacaftor and VX-561. Several sites in France were to test VX-659 but those plans have been canceled.

This week, the company also won approval for Symdeko, a combo of tezacaftor and Kalydeco, in the U.S. Analysts believe the drug could pull in $200 million this year.