Pricing, Regulatory

After a slapdown on Orkambi price, Vertex is dropping French trial sites while warning patients on access

Vertex Pharma $VRTX is playing hardball with French drug regulators — and cystic fibrosis patients waiting for their next-gen triple are getting caught in the middle.

Mired in a long-running dispute over the reimbursement level for their drug Orkambi, Vertex has decided to shut out the trial sites it had selected in France for the late-stage combination studies for its cystic fibrosis drugs, including the experimental VX-659, which has raised hopes for a broad swath of patients.

Vertex’s Orkambi was first approved in France a little more than two years ago, triggering negotiations over a reimbursement agreement as French officials pursued a deep, 80% discount based on an old drug for CF — which Vertex has bucked against. Vertex has been selling Orkambi in France at its list price as it negotiates the final price with the French government. But officials rejected that list price, saying that Orkambi provided only a “modest” benefit — an attitude that has plagued Vertex’s negotiations in Europe. The decision puts Vertex on the hook for a reimbursement off the list price, and now it’s responding by raising a warning about patient access to their drugs and shutting out French trial sites.

The company offered this statement to Endpoints News in response to a query about the trial sites:

“Our goal is to cure CF and we have made significant advances by discovering and developing the first medicines to treat the underlying cause of this disease. We want all patients to have access to these and future medicines we are developing for those still waiting. We urge the French government to join countries like the U.S., Austria, Ireland, Germany and many others to provide patients permanent access to Orkambi.”

BioCentury got hold of an excerpt of a letter Vertex sent to the labs which, they reported, stated that:

(D)espite more than 18 months of “good faith” negotiations there has been no resolution on the reimbursement of Orkambi and that therefore the pathway for CF patients in France to gain access to the drug is “unpredictable” and conducting the trials “would raise false hope.”

BioCentury got the excerpt of the communications from CF patient groups in France, which are getting caught in the middle of a fight. And it’s working in Vertex’s favor. According to the report they want Vertex to continue negotiations and get the trial sites back into the late-stage studies of the next-gen triples that promise to greatly increase the number of patients that Vertex can reach.

A couple of weeks ago Vertex picked VX-659 and VX-445 for two late-stage programs, combining each with tezacaftor and Kalydeco. Their drug ‘659 is slated to be the first off the mark after researchers demonstrated that the high dose triple improved a measure of lung function by 13.3%.


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