UK NICE to Announce Decision on Vertex’s Therapy Trikafta in December

The National Institute for Health and Care Excellence (NICE) in the U.K. is currently in the process of evaluating Vertex Pharmaceuticals’ triple combination therapy — known as Trikafta in the United States — for the treatment of cystic fibrosis (CF).

The institute expects to deliver its recommendation on Dec. 16, 2020.

NICE has the responsibility of giving advice to the National Health Services (NHS) in England on whether new therapies should be made available for physicians to prescribe. NICE’s advice is also largely followed by the health authorities in Northern Ireland and Wales.

Trikafta is a next-generation triple combination therapy — elexacaftor, tezacaftor, and ivacaftor combo — designed to address the defects in the CF transmembrane conductance regulator (CFTR) protein that underlie the disease.

In the U.S., Trikafta was approved based on results of two Phase 3 clinical trials: AURORA F/MF (NCT03525444) and AURORA F/F (NCT03525548), both of which showed that the therapy improved lung function and reduced pulmonary exacerbations in enrolled CF patients.

Currently, the European Medicines Agency (EMA), the health authority for member states in the European Union, is also assessing the triple combination therapy. Vertex expects a licence to be granted during the last three months of 2020.

Trikafta was originally under the EMA’s “Accelerated Assessment” review process, which expedites the evaluation of medicines that are of major public health interest; the process usually takes 150 evaluation days. However, to better determine the patient population that the license would cover, EMA and Vertex have extended the time of the process.

The Cystic Fibrosis Trust, the largest charity funder of CF research in the U.K., is asking for the licence to encompass anyone with CF, 12 years or older, who have at least of one copy of the F508del mutation in the CFTR gene, the most frequent CF-causing mutation.

Over the next months, the Cystic Fibrosis Trust plans to continue the conversation with EMA about Trikafta.

“While it is encouraging that the EMA and NICE planned timelines are aligned, we must continue to engage with all stakeholders, including Vertex and the NHS, to ensure a rapid, positive outcome for everyone who can benefit,” Nick Medhurst, head of policy and public affairs at the Cystic Fibrosis Trust, said in a press release.

“In Scotland, where different processes are followed to support NHS use of new medicines, this requires continuing our engagement with the Scottish Government, Scottish Medicines Consortium (SMC), and clinical teams,” he added.