Vertex, NHS England Reach Agreement on Pricing and Public Availability of CF Treatments

Vertex Pharmaceuticals has reached an agreement with England’s National Health Service (NHS), allowing patients there access to all Vertex cystic fibrosis (CF) treatments currently approved in Europe at low or no out-of-pocket cost.

The agreement between the NHS and Vertex appears to end a long-standing dispute over the company’s pricing — years in the case of Orkambi (lumacaftor/ivacaftor) — and the treatments’ inclusion in England’s public health program, the NHS.  It covers Orkambi as well as Symkevi (tezacaftor/ivacaftor), known as Symdeko in the United States, and expanded access to Kalydeco (ivacaftor).

Cystic Fibrosis Trust, a nonprofit association representing patients in the U.K., said the agreement spans two years, and is contingent on a new cost-benefit analysis of all Vertex treatments, which it expects will include Trikafta (elexacaftor, tezacaftor, and ivacaftor). This next-generation, triple combination treatment was approved in the U.S. last week and is expected to treat 90% of all with CF.

“This is a very special day and I want to thank … everyone who has been part of this campaign for their persistence and determination to keep on fighting. We know it doesn’t end here — we will continue to fight for similar access in Wales and Northern Ireland so that all people with cystic fibrosis in the UK are able to benefit from these life-saving drugs,” David Ramsden, the Trust’s chief executive, said on the group’s website.

“Also, with news that the new triple therapy, Trikafta, … could be licenced for use in the coming year, we will continue to campaign so that people with cystic fibrosis never have to wait again for the best available medicines,” Ramsden added.

Vertex was reported in the past as wanting to set Orkambi’s price at $129,000 a year (about £100,000). The terms agreed upon were not disclosed. (In the U.S., Orkambi has a list price of about $272,000 a year.)

“This important agreement, reached in collaboration and partnership with NHS England and (funding watchdog) NICE, will allow more than 5,000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease,” Ludovic Fenaux, senior vice president of Vertex International, said in a press release.

Patients with CF in England are expected to have access to these therapies under the agreement’s terms within the next 30 days, according to a Vertex press release.

CF patients, ages 2 years and older, with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (the gene defective in CF) can now be prescribed Orkambi. Those 12 years and older with either have two copies of the F508del mutation, or one copy of the F508del mutation and a copy of one of the other 14 mutations, can be prescribed Symkevi.

Both Orkambi and Symkevi help to increase the amount and function of CFTR protein on a cell’s surface.

Kalydeco will now also be available to patients ages 18 or older who have the R117H mutation, and to those starting at 12 months old who have one of the nine gating mutations.

The specific mutations for each treatment are detailed on the Vertex release.

This agreement is “fantastic news for those suffering from this terrible disease,” said Boris Johnson, the British prime minister. “I pay tribute to those who have campaigned so passionately and tirelessly on this issue — your efforts have made all the difference.”

An estimated that 10,000 people in the U.K. have CF, including 8,000 in England, Vertex said in its release.

The company said it recently reached reimbursement agreements with public health authorities in Scotland, Australia, and Spain.