Progress in US on new CF Drug and on gene therapy trials in the UK
ORKAMBI approved in the United States
The first drug to treat the underlying cause with people with Cystic Fibrosis with the most common CF gene alteration in the world f508del has been approved in the United States by the Food and Drug Administration (FDA). The drug is called ORKAMBI. Key points in relation to this drug and Ireland are:
It is aimed at People with two copies of f508del which is 56.6% of Irish CF population (654 people) although to date it is aimed at those who are aged 12 or more which is just under 50% of the CF population in Ireland (we estimate)
It has to be approved by the European Medicines Agency (EMA) and the NCPE/ HSE CPU unit in Ireland before it is made available for people with CF. It will likely undergo a Health Technology Assessment (HTA) in Ireland before they make a decision to a) approve the drug b) reject the drug or c) reject the drug on cost grounds but leaving the door open for price negotiation.
The Results of Orkambi show an increase in lung function of up to 4% (statistically significant)
Reduction in exacerbations (worsening of condition often leading to hospitalisation) of between 30 – 39%
Philip Watt, CEO of CFI stated ‘We have evidence that Orkambi has had Life-changing effects for people on clinical trials who have been in contact with us and who have reported a significant increase in quality of life. We would urge the EMA and Irish government to assess this drug as soon as possible.’
Katie Murphy, Research Officer of CFI stated ‘The promising results from the US and UK are both the outcomes of patient groups investing heavily over many years into research for innovative therapies. We wish to acknowledge the work of the Cystic Fibrosis Foundation in the US and the Cystic Fibrosis Trust in the UK in this context.’
UK Gene therapy trial results published
The results of an on-going trial into the potential use of gene therapy to improve clinical outcomes for people with CF have been published. The trial, which was primarily funded by the Cystic Fibrosis Trust in the UK, was conducted by the Gene Therapy Consortium (GTC) and shows ‘promising yet modest’ results. Scientist say there is more work to be done but believe they are on track to deliver a therapy that can make a meaningful difference to patients' lives.
Professor Eric Alton, from Imperial College London, who co-ordinated the trial, said: "there has been a significant benefit compared with placebo in cystic fibrosis patients. If a bigger study shows big benefits then it's feasible that we'll be able to offer this treatment to patients by the end of the decade."
The trial, conducted in London and Edinburgh, compared the effects of inhaled gene therapy and a placebo treatment on 136 patients with CF aged 12 and over. Over the course of a year, patients were given 12 treatments at monthly intervals.
The results, published in The Lancet Respiratory Medicine journal, showed that at the end of the trial lung function was 3.7% better in patients who received the "active" treatment. Participants with the worst lung function at the start of the study experienced a much greater 6.4% gain compared with those in the placebo group. It is important to note that these improvements and effects were inconsistent across the 62 patients in receipt of ‘active’ treatment.
The scientists are now seeking funding for the bigger "improved liposome" trial that could cost tens of millions of pounds, and would hopefully pave the way for rolling out the treatment. Professor Alton has said;
“We hope to carry out follow-up studies looking at higher, more frequent doses, combined with other treatments, and better methods for delivering the DNA into cells.”
Speaking to the BBC, Professor Stuart Elborn of Queen’s University Belfast has said “it is too soon to proceed with larger Phase-three trials” And he called for more small-scale tests to see if a larger dose would be more effective.
Philip Watt CEO of CFI stated ‘These are the results of a Phase 2 Trial which seek to prove a concept. It will take a great deal of funding and further trials at phase 2 and phase 3 level to bring this research through to a drug that can impact on people with CF. Nevertheless it is a promising development as gene therapy might impact on everyone with CF and not just those with a specific gene alteration. They will have to do further work on drug dosage and combination trails with proven CF drugs.’
3 July 2015