A new gene therapy, transformative cystic fibrosis medicines and an obesity jab included in guidance recommended during pre-election period

During the pre-election period, 22 May to 5 July 2024, restrictions imposed on public bodies meant NICE was prevented from publicising its work. We did still continue developing useful and useable guidance to get the best treatments to patients fast, and in fact published more than 20 pieces of draft and final guidance.

On 4 June, final guidance recommending once-a-day tablet ivosidenib with azacitidine (also called Tibsovo and made by Servier Laboratories) for untreated acute myeloid leukaemia with an IDH1 R132 mutation. Around 130 people can now benefit from this new treatment option.

We launched a consultation on our committee’s draft recommendations to offer people with a BMI of 35+ and at least one weight-related comorbidity the weight loss medication tirzepatide alongside a reduced-calorie diet and increased physical activity on June 4.

Around 4,000 people with sickle cell disease (SCD) can now benefit from a new treatment we recommended in final guidance published on 12 June. 

Voxelotor (also called Oxbryta and made by Pfizer) treats haemolytic anaemia caused by SCD and is recommended for use on the NHS following a reduction in its price.

On 19 June we published final guidance recommending tafamidis for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM).  

There are around 800 people in the UK with ATTR-CM. A progressive, life limiting and debilitating condition, it is characterised by the abnormal build-up of a protein called amyloid, primarily in the heart. Symptoms start in adulthood and get worse over time, causing the heart tissue to thicken and stiffen. ATTR-CM can lead to heart failure, but until now treatment options were limited to managing symptoms and best supportive care.

Taken as a once-a-day tablet, tafamidis (also called Vyndaqel and made be Pfizer) is the first medicine for ATTR-CM that aims to treat the disease. 

On 20 June we published final draft guidance recommending cystic fibrosis (CF) treatments Kaftrio, Symkevi and Orkambi following a new commercial deal agreed between NHS England and company Vertex.

It means around 8,200 people already having these treatments in England will continue to get them routinely on the NHS along with people who are diagnosed with CF in the future. The commercial agreement between NHS England and Vertex also includes access to any future licence extensions of these medicines.

NICE, NHS England and Vertex have also committed to work together on a path towards rapid access for all eligible patients for the next-in- class triple combination treatment for CF, the investigational therapy vanzacaftor/tezacaftor/deutivacaftor, subject to authorisation by the Medicines and Healthcare products Regulatory Agency (MHRA).

NICE’s appraisal of cystic fibrosis medicines is one of the first times non-cancer medicines have received additional weighting with the new severity modifier and is a key reason why NICE has been able to recommend them.

On 27 June etranacogene dezaparvovec (also called Hemgenix and made by CSL Behring) became the first gene therapy to be recommended via our Technology Appraisal programme, the first treatment for haemophilia we have recommended and the first pilot for the outcomes-based scheme as part of the commitments in the 2024 VPAG.

Our final draft guidance recommending the treatment for around 260 adults with moderately severe or severe haemophilia B meant it also became the first medicine to go into the Innovative Medicines Fund.

The committee concluded the one-off infusion, the world’s first gene therapy for the bleeding condition, could be recommended for use on the NHS while further data is collected, particularly on how long its effect last.

On 18 June we opened public consultation on the draft joint asthma: diagnosis, monitoring and chronic asthma management guideline. 

For the first time, the NICE, British Thoracic Society (BTS), and the Scottish Intercollegiate Guideline Network (SIGN) have worked together to produce new UK-wide joint guidance for the diagnosis and management of chronic asthma in adults, young people, and children.  The consultation on the draft guideline will be open until Tuesday, 30 July and the final guideline is due to publish later this year.