2:23 PM
Aug 09, 2018
 |  BC Extra  |  Company News

Second Vertex drug caught in CF standoff

NICE's suspension of its review of Symkevi tezacaftor/ivacaftor on Thursday marks the latest delay of access in England and Wales to cystic fibrosis drugs from Vertex Pharmaceuticals Inc. (NASDAQ:VRTX).

NICE suspended its review after the company did not provide an evidence submission, according to the committee's website. It also canceled plans to discuss Symkevi's appraisal at a Nov. 8 meeting.

EMA's CHMP recommended approval of Symkevi last month.

In a statement emailed to BioCentury, David Ramsden, chief executive of the U.K.-based Cystic Fibrosis Trust, said Vertex not submitting evidence to NICE, "is awful news for thousands of people in the U.K. with cystic fibrosis and it is a situation that I find deeply concerning."

Vertex spokesperson Heather Nichols said the company believes that "NICE’s single technology appraisal has not kept pace with changes in medicine and has significant limitations in how it captures and values the full benefits of precision medicines such as tezacaftor/ivacaftor."

"We would be happy to re-engage with NICE in the context of an appropriate appraisal process, which takes into account the benefits our cystic fibrosis medicines provide to people with this devastating disease," she said.

This adds a second drug to Vertex's clash with NICE over reimbursement of its CF drugs in England and Wales. In 2016, NICE issued final guidance saying Orkambi lumacaftor/ivacaftor was not cost-effective. Several European countries, including the U.K. and France, have yet to reach a reimbursement agreement for Orkambi.

At a parliamentary debate in March, several MPs appeared to share Vertex's concerns over the NICE appraisal process, saying that it was not appropriate for appraising rare disease drugs like Orkambi and that the next government review of NICE should consider whether the agency's methods are appropriate for rare diseases. The debate was held in response to a public petition asking that the government resolve the CF drug pricing dispute (see "MPs Call on U.K. Government to Resolve Orkambi Impasse").

Last month, Vertex EVP and Chief Commercial Officer Stuart Arbuckle said the company would not be accepting NHS England's July 20 counteroffer regarding pricing for the company's CF portfolio. Under NHS England's final proposal, it would pay Vertex about £500 million ($662 million) over the next five years, and over £1 billion ($1.3 billion) over the next 10 years (see "NHS Makes 'Final Offer' to Vertex").

Nichols told BioCentury on Thursday that Vertex Chairman, President and CEO Jeff Leiden requested to meet with Head of NHS Simon Stevens and Secretary of State for Health and Social Care Matt Hancock several weeks ago to continue negotiations related to Orkambi as well as its next-generation therapies, but has not heard back.

"Next week marks 1,000 days since it was licensed in the U.K.,” said Ramsden of Orkambi. “Yet it is still not available on the NHS."

FDA approved Symkevi as Symdeko in February. It is a fixed-dose tablet combination of tezacaftor, a small molecule corrector of cystic fibrosis transmembrane conductance regulator (CFTR), and ivacaftor, a small molecule potentiator of CFTR.

Vertex lost $0.25 to $175.05 Thursday.

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