12:07 PM
Feb 14, 2018
 |  BC Extra  |  Company News

Vertex cancels CF trials in France

French cystic fibrosis patients may not receive access to treatments under development by Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) after the company canceled French sites for three planned Phase III trials slated to evaluate combinations of the biotech's CF transmembrane conductance regulators (CFTRs).

Although Vertex did confirm the cancellations to BioCentury, the company declined to offer an explanation. Three French CF associations told BioCentury that Vertex’s decision came after the company reached a stalemate in reimbursement negotiations for its CF drug Orkambi ivacaftor/lumacaftor with the French government. The groups -- Overcoming Mucoviscidosis, the Grégory Lemarchal Association and the French Cystic Fibrosis Society -- provided BioCentury with an excerpt from what they described as an official explanation sent by Vertex on Feb. 5 to French laboratories that had previously been retained to participate in the trials, which said that despite 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."

Orkambi gained EU approval in November 2015 to treat CF patients ages 12 and older who have two copies of the delta F508 mutation; however the company has not agreed upon a definitive reimbursement price with the French government. Vertex has been invoicing the government for supplies of the drug based on the company's proposed list price; if French authorities do not accept Vertex's proposed price, the company will have to refund any overpayment. According to the CF groups, the government recently denied the proposed price after determining Orkambi's benefit to be "modest."

Orkambi is still available to French CF patents ages 12 and older with two copies of the delta F508 mutation, the CF groups told BioCentury. In January, Orkambi's EU approval was extended to CF patients ages 6-11 who have two copies of the delta F508 mutation. According to the letter, Vertex said the pathway for additional CF patients to gain access to the biotech's treatments "is unpredictable and the outcome uncertain."

The CF organizations said the canceled French sites were to participate in two upcoming Phase III trials to evaluate triple combinations of Vertex's CFTRs -- ivacaftor, tezacaftor and VX-659 -- in patients 12 and older with two copies of the delta F508 mutation; and in patients 12 and older with one copy of the delta F508 mutation. The third Phase III trial with canceled French sites is to evaluate ivacaftor plus tezacaftor in patients ages 6-11 with one or two copies of the delta F508 mutation. Last month, Vertex announced it would begin the three trials this half. Other European countries will participate in the trials as planned. VX-659 is a next-generation CTFRs.

Vertex said French sites could also be excluded from a fourth Phase III trial to evaluate ivacaftor and tezacaftor plus another next-generation CTFR, VX-445. That program is slated to begin mid-year.

The three CF groups called on Vertex to reconsider its position concerning the planned clinical trials and urged it to continue its pricing negotiations.

Kalydeco ivacaftor, the first of Vertex's marketed CF drugs, has been available in France since 2012.

An MAA for Vertex's Symdeko tezacaftor/ivacaftor is under EMA review, with a decision expected next half. On Monday, FDA approved an NDA for the drug to treat cystic fibrosis in patients ages 12 and older who have two copies of the delta F508 mutation or who have at least one CF gene mutation that is responsive to Symdeko (see BioCentury Extra, Feb. 12).

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