Teriflunomide (Aubagio)

Transcription

1 Teriflunomide (Aubagio) Spirella Building, Letchworth, SG6 4ET reg charity no

2 We hope you find the information in this factsheet helpful. If you would like to speak with someone about any aspect of MS, contact the MS Trust information team and they will help find answers to your questions. This factsheet has been provided free by the Multiple Sclerosis Trust, a small UK charity which works to improve the lives of people affected by MS. We rely on donations, fundraising and gifts in wills to be able to fund our services and are extremely grateful for every donation received, no matter what size. MS Trust information service Helping you find the information you need The MS Trust offers a wide range of publications, including a newsletter Open Door, which provides an ongoing update on research and developments in MS management. In addition it contains articles from people with MS and health professionals. For a full list of MS Trust publications, to sign up for Open Door and much more visit our website at Freephone (Lines are open Monday - Friday 9am-5pm) write infoteam@mstrust.org.uk MS Trust Spirella Building Letchworth Garden City SG6 4ET

3 Teriflunomide (Aubagio) Date of issue: January 2014 This factsheet will be reviewed within three years Contents 1. Overview 1 2. How does it work? 2 3. How is it taken? 2 4. What are the results so far? 2 5. Side effects 3 6. What further studies are planned? 3 7. Licensing and appraisal 3 8. References 3 9. Drug development process explained 4 1. Overview Drug in development Teriflunomide Other names What is it? Aubagio, HMR1726, A Teriflunomide is a drug treatment for relapsing remitting multiple sclerosis Teriflunomide is taken as a tablet, once daily. Teriflunomide reduces the number of white blood cells (T-cells and B-cells) involved in the MS immune response. In phase III studies, people taking teriflunomide had about one third fewer relapses than people taking placebo (dummy pill). The most common side effects have been: nausea and diarrhoea increased liver enzyme levels hair thinning NICE (National Institute for Health and Care Excellence) has approved teriflunomide as an NHS treatment for active relapsing remitting MS in England and Wales. SMC (Scottish Medicines Consortium) has met to consider use by the NHS in Scotland, with a decision expected in February

4 2. How does it work? In multiple sclerosis, the immune system mistakenly attacks the myelin sheath that surrounds nerve cells in the brain and spinal cord. The damage disrupts the way in which messages, or nerve impulses, are carried to and from the brain, leading to a variety of symptoms. The mechanism of action of teriflunomide is not completely understood but it is thought that the main effect is to stop certain immune cells from multiplying 1. This results in lower numbers of both B-cells and T-cells, two types of white blood cells involved in the immune response associated with MS. 3. How is it taken? Teriflunomide is taken orally as a tablet, once daily. 4. What are the results so far? Phase III studies indicate that teriflunomide reduces relapses by about one third compared to placebo. TEMSO 2 - this phase III, two year study compared two doses of teriflunomide with placebo in 1088 people with relapsing remitting MS. Both doses of teriflunomide reduced the number of relapses by 31% compared to placebo. The higher dose (14mg daily) also reduced disability progression by 30% compared to placebo. TENERE 3 - compared two different doses of teriflunomide with interferon beta 1a in 324 people over two years and measured how long it took till participants had a relapse; there was no difference in this measure between the three groups. Two further phase III studies have been completed but have not been published in a peer-reviewed journal. Details of the studies have been presented at scientific meetings. The TOWER study tested two different doses of teriflunomide or placebo for at least 48 weeks in 1169 participants. The higher dose (14mg) reduced relapses by 36% compared to placebo and reduced disability progression by 31%. The TOPIC study investigated whether treating people early on, when they first experience 2

5 an episode of MS-like symptoms (also known as clinically isolated syndrome, CIS), will delay the onset of confirmed MS. Those taking teriflunomide were less likely to develop confirmed MS during the course of the two year study compared to the group taking placebo. 5. Side effects The most common side effects include: nausea and diarrhoea. increased liver enzyme levels. hair thinning and loss. 6. What further studies are planned? No further clinical trials are currently underway. 7. Licensing and appraisal The manufacturer submitted teriflunomide for licensing in Europe in February In August 2013, the European Commission approved teriflunomide as a treatment for adults with relapsing remitting MS. In January 2014, NICE (National Institute for Health and Care Excellence) published guidance for the NHS in England and Wales, approving teriflunomide as a treatment for adults with active relapsing remitting multiple sclerosis (normally defined as 2 clinically significant relapses in the previous 2 years). NICE has not approved teriflunomide for people with highly active or rapidly evolving severe MS. SMC (Scottish Medicines Consortium) is reviewing teriflunomide for use by the NHS in Scotland with a decision expected in February References 1. Claussen MC, et al. Immune mechanisms of new therapeutic strategies in MS - teriflunomide. Clinical Immunology 2012;142: O'Connor P, et al. Randomized trial of oral teriflunomide for relapsing multiple sclerosis. New England Journal of Medicine 2011;365: Vermersch P, et al. Teriflunomide versus subcutaneous interferon beta-1a in patients with relapsing multiple sclerosis: a randomised, controlled phase 3 trial. Multiple Sclerosis 2013 Nov 21. [Epub ahead of print] Please contact the MS Trust Information Team if you would like any further information about reference sources used in the production of this publication. 3

6 9. Drug development process explained Phase I The first step in testing a new drug is to determine the safety of single doses in a small number of healthy volunteers. This stage helps researchers understand some aspects of how it works and establishes the likely dose required. Phase II If the treatment proves to be safe, studies begin to determine the effectiveness of the drug in people with the condition to be treated. These studies may last several months or years and involve larger numbers of people, perhaps one or two hundred. The study will generally be controlled - the drug is compared with the standard treatment or placebo (dummy treatment) double-blind - neither the investigators nor the participants know which treatment they are receiving, and randomised - participants will be randomly allocated to receive active treatment or placebo. Phase III If a drug shows effectiveness, a larger study is conducted in hundreds of people. These clinical trials may be conducted at different locations (multi-centre) and across several countries and may last several years. These studies allow researchers to more accurately assess the potential of the new drug in a wider range of people and compare it to existing treatments Licensing Data from all of these phases is presented to the regulatory authorities (in the UK this is the MHRA - Medicines and Healthcare products Regulatory Agency or the EMA - European Medicines Agency). If the authorities are satisfied that the new medicine is effective, safe and meets manufacturing quality standards, a marketing authorisation or licence is issued. NHS appraisal Once a new medicine has been licensed, there may still be one final hurdle. Set up in 1999 to help eliminate postcode lottery prescribing in England and Wales, NICE (National Institute for Health and Care Excellence) appraises certain new medicines and will look at issues such as cost effectiveness of the new treatment. In Scotland, appraisal is carried out by the SMC (Scottish Medicines Consortium). The NHS is legally obliged to fund medicines recommended by NICE or SMC. If NICE or SMC do not approve a new medicine, then local health authorities are not required to provide funding for it. 4