Medserena Upright Open MRI centres in London and Manchester offer patients a state-of-the-art MRI examination that removes the feeling of claustrophobia.
Conventional MRI scanners require them to slide into a narrow tunnel which many people find uncomfortable and cramped inducing anxiety or panic. Even an ‘open’ MRI scanner still requires them to endure the machine very close to their face.
With the Medserena Open MRI scanner, the patient can stand up, sit down, flex their neck and be moved into different postures – so the scan is carried out in exactly the position that pain is experienced.
All coils (antennae), even the head coils for brain scans, are designed to allow patients to clearly see outside the system. A large TV screen allows them to watch a TV programme or DVD whilst the process is carried out.
GW Pharmaceuticals plc presented a variety of data on cannabidiol oral solution (CBD) at the 13th Annual European Congress of Epileptology (ECE), which took place in Vienna, Austria from 26-30 August 2018.
The studies provide additional insight into the safety and efficacy of GW’s CBD oral solution in the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome, two rare and severe forms of childhood-onset epilepsy that are highly treatment-resistant1,2. In addition, data related to the pharmacology of CBD, and its antiseizure properties, will provide additional understanding of CBD’s role in the management of such diseases. A marketing authorisation application for GW’s CBD oral solution is currently under review by the European Medicines Agency (EMA).
“GW is proud to be presenting a wealth of data on its CBD oral solution to the epilepsy community. Our comprehensive pre-clinical and clinical programmes provide an understanding of the way in which CBD exhibits anti-seizure effects and further supports its efficacy and tolerability profile in patients living with two of the most difficult-to-treat forms of epilepsy,” stated Justin Gover, GW’s Chief Executive Officer. “There is still a huge unmet medical need for effective medicines that help people suffering from severe forms of treatment resistant epilepsy, and we believe that our CBD oral solution may present an important new therapeutic option in the future.”
CBD data highlights:
Highlights include the presentation of pooled efficacy and safety data from two phase III randomised placebo-controlled trials of CBD in LGS, Phase 1 drug-drug interaction data on the co-administration of CBD and clobazam, and the potential role of GPR55 and the TRPV1 receptor-dependent interaction in the anti-epileptic properties of CBD.
The full list of GW titles presented is as follows:
Cannabidiol (CBD) Significantly Reduces Drop Seizure Frequency in Lennox-Gastaut Syndrome (LGS): Pooled Efficacy and Safety Results from 2 Randomized, Controlled Trials
Bidirectional Drug-drug Interaction with coadministration of Cannabidiol and Clobazam in a Phase 1 Healthy Volunteer Trial
Maintained Safety and Efficacy of Cannabidiol (CBD) in a Long-Term Open-Label Trial in Patients with Lennox-Gastaut Syndrome (LGS) (GWPCARE 5)
Exposure-Response Analysis of Cannabidiol Oral Solution for the Treatment of Lennox–Gastaut SyndromeA Phase 2 Trial to Explore the Potential for a Pharmacokinetic Drug-drug Interaction with Valproate when in Combination with Cannabidiol in Adult Epilepsy Patients (late breaking abstract)
Antiseizure properties of CBD are attenuated in the absence of TRPV1 receptors
A role of GPR55 in the anti-epileptic properties of cannabidiol
Maintenance of Long-Term Safety and Efficacy of Cannabidiol (CBD) Treatment in Dravet Syndrome (DS): Results of the Open-Label Extension (OLE) Trial (GWPCARE 5)
Bidirectional Drug-drug Interactions with Co-administration of Cannabidiol and Stiripentol or Valproate in a Phase 1 Healthy Volunteer Trial
Phase 3 TOLEDO study shows apomorphine infusion provides effective relief of persistent motor fluctuations in Parkinson’s disease (PD) patients whose symptoms are uncontrolled with oral medication
Britannia Pharmaceuticals Ltd has announced publication of the results of the double-blind phase of the TOLEDO study in Lancet Neurology1.
Treatment with APO-go®/MOVAPO® (apomorphine) subcutaneous infusion for 12 weeks gave significantly greater reductions in OFF time (periods when PD medications don’t work) from baseline compared with placebo: –2.47 h/day versus –0.58 h/day, respectively – a treatment difference of almost 2 hours (p=0.0025) and double the change in OFF time recognised as meaningful to PD patients. These reductions were seen in the first week of treatment with APO-go®/MOVAPO® infusion.
Compared with placebo, significantly greater increases in ON time (periods with good motor control) without troublesome dyskinesia – ‘good’ ON time – from baseline were observed with APO-go®/MOVAPO® infusion: 2·77 h/day versus 0·80 h/day, respectively (p=0·0008), and patients could also reduce the dosage and number of administrations of concomitant oral PD medications significantly (p=0.0014).
APO-go®/MOVAPO®/ infusion is an established therapy for PD, a disease characterised by progressive degeneration of dopamine-containing neurons in the brain, resulting in loss of motor control. TOLEDO is the first multicentre, randomised, double-blind trial to investigate its efficacy and safety in PD and was undertaken in 107 patients from 23 hospitals in 7 countries whose symptoms were uncontrolled despite taking multiple medications.
Clinical improvements were reflected in patients’ assessment of treatment: significantly more APO-go®/ MOVAPO® infusion patients rated themselves as ‘improved’ (71%) versus placebo (18%; p<0.0001).
Professor Regina Katzenschlager, lead investigator of TOLEDO, commented:
TOLEDO is an important addition to our knowledge, providing Level 1 evidence for the first time and confirming previous observational studies. Apomorphine infusion is effective and well tolerated by patients experiencing debilitating treatment response fluctuations despite optimised treatment.
Professor Andrew Lees, an investigator in the pivotal clinical trial that led to apomorphine being licensed for PD treatment in the UK, added:
We hope the positive results of the TOLEDO study will help ensure apomorphine infusion, which is delivered using a small, ambulatory mini-pump, is incorporated into national PD treatment guidelines.
The TOLEDO study is sponsored by Britannia Pharmaceuticals Ltd., part of the STADA Arzneimittel AG group of companies and manufacturer of apomorphine products.
Rett UK is the only UK charity providing professional support to families affected by the rare neurological disorder, Rett syndrome; a severe, life long, life limiting genetic neurological disorder and the most common cause of profound learning disabilities affecting mainly females (1 in 12,000).
Although present at birth, it is usually undetected until a major regression occurs at around two years of age, when children lose acquired skills and the complexity of the disability is revealed.
Rett UK provides emotional and practical support through a national telephone helpline, local family led support groups and a parent-to-parent contact network all so crucial in reducing isolation. Access to high quality seminars from the UK’s leading experts on Rett syndrome at our regional events provides families and professionals with up to date, accurate information in subjects like epilepsy, spinal surgery and communication, helping them with management of the disability.
European Medicines Agency PRIME (PRIority MEdicines) status is granted to medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options
RG6042 has the potential to be the first therapy targeting the underlying cause of Huntington’s disease, a fatal neurodegenerative rare disease
Third PRIME designation for a Roche medicine
Roche today announced that the European Medicines Agency (EMA) has granted PRIME (PRIority MEdicines) designation for the company’s investigational medicine RG6042 (formerly known as IONIS-HTTRx) for the treatment of people with Huntington’s disease (HD). RG6042 has demonstrated its ability to reduce the toxic mutant huntingtin protein (mHTT), which is believed to be the underlying cause of HD, in a Phase I/IIa study.1 PRIME is a designation implemented by the EMA to support data generation and development plans for promising medicines, providing a pathway for accelerated evaluation by the agency, and thus potentially enable them to reach patients earlier.2
“We are very pleased that the European Medicines Agency has granted PRIME designation for RG6042, as there is an urgent medical need to find treatment options for families affected by Huntington’s disease,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “Preliminary data on RG6042 were the first to show that levels of toxic mutant huntingtin protein can be lowered in adults with Huntington’s disease, and we are working closely with the EMA and other health authorities to initiate a global phase III study as soon as possible.”
PRIME designation for RG6042 is primarily based on the data from an exploratory Phase I/IIa trial of RG6042 that demonstrated a significant reduction in mHTT, which breaks down the nerve cells in the brain.1 The study demonstrated a mean 40% (up to 60%) reduction of the specific HD protein in the cerebrospinal fluid (CSF) of adult patients treated with RG6042 for three months at the two highest doses. Furthermore, levels of mHTT measured in the CSF were still declining in the majority of treated patients (~70%) as of the last measurement in the study.3 RG6042 was well tolerated in this short initial study.1 These data were shared at the CHDI 13th Annual HD Therapeutics Conference in March 2018,3 and updated results were presented at the American Academy of Neurology (AAN) Annual Meeting in April 2018.4
Roche will initiate a pivotal phase III study to evaluate RG6042 in a larger patient population to further characterise the safety profile and determine if it can slow the progression of HD in adults.
About RG6042
RG6042 is a second-generation modified antisense oligonucleotide (ASO) designed to reduce the production and levels of mHTT protein by targeting human HTT mRNA.5 RG6042 is the result of a comprehensive drug discovery programme between Roche and Ionis Pharmaceuticals focused on optimising the potency, specificity and tolerability of an ASO targeting human HTT mRNA. RG6042 is the most advanced compound in clinical development to target toxic mutant huntingtin protein (mHTT), which is believed to be the underlying cause of HD. Treatment with RG6042 has the potential to slow or stop disease progression in all people with HD.1
About Huntington’s disease
Huntington’s disease is a rare genetic, progressive condition that causes the nerve cells in the brain to break down, which severely affects a person’s everyday functions such as mobility and thinking.6 It has a devastating impact on people living with the disease, and the hereditary nature of HD means it profoundly affects entire families.6 As the disease progresses, people with HD may develop personality changes, difficulty walking and swallowing, as well as having a significant cognitive impact.6 Survival ranges from approximately 10-20 years following motor onset of the disease.6
There is no known cure for HD and no approved therapies that treat the underlying cause. The estimates for the number of people affected by Huntington’s vary between geographic regions. Huntington’s disease is the most common monogenic neurological disorder in the developed world, with an estimated prevalence of 12.3/100,000 in the United Kingdom, equating to ~8,000 people.7
About Roche in neuroscience
Neuroscience is a major focus of research and development at Roche. The company’s goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, Alzheimer’s disease, spinal muscular atrophy, Parkinson’s disease, Huntington’s disease and autism spectrum disorder.
About Roche Products Limited
Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology and infectious diseases. Roche is also the world leader in in–vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Roche’s personalised healthcare strategy aims at providing medicines and diagnostics that enable tangible improvements in the health, quality of life, safety and survival of patients. Twenty-eight medicines developed by Roche are included in the WHO Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and chemotherapy. Roche employs over 2,000 people in pharmaceuticals and diagnostics in the UK.
All trademarks used or mentioned in this release are protected by law.
Adverse event reporting
Adverse events should also be reported to Roche Products Ltd. Please contact Roche Drug Safety Centre by emailing welwyn.uk_dsc@roche.com or calling +44(0)1707 367554.
References
Ionis Pharmaceuticals. 2018. Press release: IONIS-HTT Rx (RG6042) top-line data demonstrate significant reductions of disease-causing mutant Huntingtin protein in people with Huntington’s disease. [Internet; cited 2018 July]. Available from: http://ir.ionispharma.com/node/23401/pdf.
Ionis Pharmaceuticals. 2018. Press release: New Data from IONIS-HTT Rx Phase 1/2 Study Demonstrates Correlation Between Reduction of Disease-causing Protein and Improvement in Clinical Measures of Huntington’s Disease. [Internet; cited 2018 July]. Available from: http://ir.ionispharma.com/node/23661/pdf.
Leavitt B, Tabrizi S, Kordasiewicz H et al. Discovery and early clinical development of ISIS-HTTRx, the first HTT-lowering drug to be tested in patients with Huntington’s disease (PL01.002). Neurology 2016;86(Suppl. 16):PL01.002. [Internet; cited 2018 July]. Available from: http://n.neurology.org/content/86/16_Supplement/PL01.002.
The UK’s oldest surgical Royal College is switching gears and taking to the road, on the first-ever ‘Cycling Symposium’ across the UK.
Pictured: Giles Bond-Smith, Katherine Hurst and Mike Silva
On behalf of the 500+ year-old Royal College of Surgeons of Edinburgh (http://www.rcsed.ac.UK), a dozen surgeons and surgeons-in-training will be setting off on 1 September on a gruelling seven-day bicycle trek from Southampton to Edinburgh – in a bid to spread the word on the latest news and advances in the field of surgery.
On this mission, the team of experienced consultants and talented trainees will stop each evening to conduct a series of masterclasses on key surgical topics. These include highlighting the importance of patients’ cardiovascular fitness prior to surgery, an update on the College’s groundbreaking anti-bullying campaign #LetsRemoveIt, as well as other subjects surrounding safety in the operating theatre, reducing the risk of infections, ‘never events’ and topics of special interest in vascular surgery and trauma surgery.
The team, led by Oxford-based Consultant Liver, Pancreatic and General Surgeon and RCSEd Deputy Surgical Director of the Regional Advisory Network, Mr Mike Silva and Academic Surgical Fellow at Oxford University Hospital and Member of the RCSEd’s Trainees’ Committee Miss Katherine Hurst, will set off from Southampton, pedalling through Oxford, Birmingham, Sheffield, Middleborough, Newcastle and Carlisle to arrive in Edinburgh after a journey of almost 550 miles. Keen cyclists are invited to take part too, by joining the surgeons and cycling part of the route with them.
Spearheading the cycling effort Mr Mike Silva said:
“Poor physical fitness can lead to costly yet avoidable complications after surgery and there is strong evidence that improving a patient’s functional capacity lends to better surgical outcomes. In October 2014, the RCSEd launched a UK-wide education campaign to get patients exercising adequately prior to their surgery. A key aspect of the ‘Cycling Symposium’ will therefore highlight the positive aspects of physical activity like cycling.
“#LetsCycleIt will also incorporate a series of surgical masterclasses each evening. The topical discussions will be delivered by surgeons who have taken part in the ride to a diverse audience that will include members of the public, medical students, junior doctors in training and practicing surgeons.”
Cycling alongside Mr Silva will be Miss Katherine Hurst, an Academic Surgical Fellow at Oxford University Hospital:
“Over 80% of the College’s UK members are based in England and Wales, so we’re taking to the roads to highlight the presence of RCSEd throughout Britain, and the work undertaken to improve patient outcomes and ensure surgery is a profession that remains thriving and inclusive. The College has been championing programmes such as the hugely successful national anti-bullying campaign #LetsRemoveIt, and showcasing roles for women in surgery. Studies estimated that bullying within the NHS costs UK organisations nearly £14billion per year, and healthcare professionals have attributed disruptive behaviour alone to 67% of adverse patient events, 71% of medical errors, and 27% of perioperative deaths. Bullying and harassment in the workplace is no longer acceptable and our travelling symposium will inform and equip surgeons to tackle this detrimental behaviour.
“Women form an ever increasing proportion of the UK’s surgical workforce and the College has an increasing number of female trainees, surgeons and Council members. A number of female surgeons will be participating as riders and will showcase opportunities for women choosing a career in surgery.”
The #LetsCycleIt route will be open to local cyclists, both healthcare professionals and the public. To join in any of the seven daily stages, register online beforehand via the RCSEd website.
The President of the Royal College of Surgeons of Edinburgh Professor Mike Lavelle-Jones said: “I wish Mike and Katie, along with all the other cyclists, a most successful trip and I look forward to meeting up with them from time to time along the way. This is a unique event, which provides an opportunity to emphasise the importance of fitness, not only to our patients but also to the population at large. The cycle ride across the heart of England is a bold initiative and I applaud all of the team for their sterling efforts.”
Young Epilepsy and Veriton Pharma Ltd (Veriton) have announced a key agreement, where the organisations will work in partnership to deliver the ‘Rules 4 Schools” element of the charity’s newly launched ‘#InTheMoment’ initiative.
Through this policy influencing initiative, Young Epilepsy is looking to break down the obstacles to opportunity for the 55,800 school aged children (5-18 years) with epilepsy in the UK1. Starting with the ‘Rules 4 Schools’ campaign, the key aim for the charity, supported by Veriton, is to challenge current thinking and encourage systemic change in priority areas.
Since 2014, all state schools in England are legally required to have a policy on supporting children with medical conditions such as epilepsy. This means that all children with epilepsy should have an Individual Healthcare Plan (IHP) which sets out essential information such as how the condition affects them and what to do in an emergency. However, Young Epilepsy believe many schools have some way to go in adequately supporting children with epilepsy.
Schools need to make sure that children with epilepsy have the support they need to fulfil their potential in an inclusive and safe environment. In a survey2 we carried out in 2017, we found that 1 in 3 (36%) of young people with epilepsy still don’t have an IHP at school and only 51% of families surveyed said that school staff had been trained to support a young person with epilepsy. This needs to be dramatically improved and we think the ‘Rules 4 Schools’ can help to achieve this.”
Matt Robertson, Young Epilepsy
The ‘Rules 4 Schools’ campaign will concentrate on four key areas:
Encouraging or working with schools to ensure all young people with epilepsy have an individual healthcare plan
Encouraging or working with schools to have a policy on supporting pupils with medical conditions
Achieving a requirement on schools to publish medical conditions policies on their websites
Campaigning for school inspections to include a routine check for support for pupils with medical conditions
We are delighted to be working in partnership with Veriton on this critically important campaign and we really look forward to making a difference to as many young people with epilepsy as possible through the #InTheMoment – Rules 4 Schools initiative.”
Young Epilepsy is a member of the Health Conditions in Schools Alliance which is made up of over 30 organisations including charities, healthcare professionals and trade unions working collaboratively to make sure children with health conditions get the support they need at school.
Young Epilepsy is the only national charity with the sole purpose of supporting children and young people aged under 25 with epilepsy and associated conditions, as well as their families. The charity has been improving the lives of children and young people living with epilepsy and related neurological conditions for over 120 years with the aim of enabling them to fulfil their potential and ensure they have the best quality of life. Currently in the UK, there are around 112,000 young people with epilepsy of which 55,800 are school aged children (5-18 years)1.
About Veriton Pharma Ltd
Founded in 1997 by Dr Graham March MrPharmS, the company has pioneered the development and availability of innovative products and specialised medicines in the fields of epilepsy, neurology and rare paediatric conditions. For more information visit http://www.veritonpharma.com
Research presented at The Alzheimer’s Association International Conference (AAIC) recently shows that “Z-drugs” such as zolpidem, zopiclone and zaleplon, often prescribed to treat insomnia, could lead to an increase in falls for people with dementia.
Researchers from the Norwich Medical School University of East Anglia found that the use of Z-drugs is associated with a 40% increase in any type of fractures, with this risk increasing with higher doses of the medication.
Sleep disturbances are a common symptom of dementia, and can make life for the person with dementia, and their carer, really challenging. To tackle this, we’re funding research to better understand the importance of sleep in brain health. However, the finding in this study that people who had taken Z-drugs to sleep were more prone to falls and injuries as a result echoes current research. We recommend personalised non-drug solutions, like avoiding caffeine or taking exercise in the day, to help with sleep.With dementia prevalence rising – the number is set to be 1 million by 2021 – only research will beat this devastating condition for good.
Dr Doug Brown, Chief Policy and Research Officer at Alzheimer’s Society
Read ACNR’s article, “Falls as a result of being on Z-drugs for insomnia” in the May-July 2018 issue.
Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc., have announced that TegsediTM (inotersen) has received marketing authorisation approval from the European Commission (EC) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). This follows the positive opinion recommending approval provided by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).1
“With the EC’s decision, inotersen is now the world’s first and only RNA-targeted therapeutic approved for patients with hATTR amyloidosis. With subcutaneous delivery, inotersen puts treatment in the patients’ hands while bringing the significant benefits shown in the NEURO-TTR study in both measures of neuropathy and quality of life for people living with this serious and fatal disease. This is an important day for the hATTR amyloidosis community as we believe inotersen enables people and their families impacted by this disease to move forward with their lives,” said Paula Soteropoulos, chief executive officer at Akcea Therapeutics. “Today is a milestone for Akcea with our first drug approval. It is an achievement we share with the courageous hATTR patient community in Europe and around the globe. We are ready to launch inotersen along with our patient and physician support services across Europe.”
The abnormal formation and aggregation of transthyretin (TTR) protein results in TTR amyloid deposits throughout the body and is the underlying cause of hATTR amyloidosis. Inotersen is designed to block production of the TTR protein. In the NEURO-TTR study, treatment with inotersen produced substantial reductions in the levels of the TTR protein regardless of mutation type or stage of disease.2
“hATTR amyloidosis is an inherited, progressive and fatal disease for which treatment options are limited. The approval of inotersen brings us into a new era of treatment with an efficacious and disease modifying medicine that potentially allows patients to achieve a greater degree of independence,” said Teresa Coelho, M.D., neurologist and neurophysiologist at Santo António Hospital, Porto, Portugal. “Inotersen has demonstrated rapid and sustained benefits in improving the course of this disease and preserving quality of life.”
The European Commission’s approval of inotersen was based on results from the Phase 3 NEURO-TTR study in patients with hATTR amyloidosis, with symptoms of polyneuropathy.2 Results from that study demonstrated that patients treated with inotersen experienced significant benefit, compared to patients treated with placebo across both co-primary endpoints: the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression.2 Inotersen is associated with risk of thrombocytopenia and glomerulonephritis. Enhanced monitoring is required to support early detection and management of these identified safety risks.2
“Today, we are thrilled to see our successful research and development efforts result in the approval of an important new medicine for patients with hATTR amyloidosis. Using our antisense technology platform, we set out to design a therapy to block the production of the underlying cause of this disease, the TTR protein,” said Brett P. Monia, Ph.D., chief operating officer at Ionis Pharmaceuticals. “Approval of inotersen further establishes Ionis as a multi-product company. We are confident that the experienced team at Akcea will deliver on the promise of inotersen. We are grateful to all of the physicians and patients who participated in the inotersen clinical program and who made this landmark approval possible.”
For important safety information for inotersen, including method of administration, special warnings, drug interactions and adverse drug reactions, please see the European Summary of Product Characteristics (SmPC), available from the EMA website at www.ema.europa.eu.
Inotersen is also under regulatory review in the United States and Canada. Inotersen’s U.S. Prescription Drug User Fee Act, or PDUFA, date is October 6, 2018.
ABOUT INOTERSEN
Inotersen is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production. Inotersen is approved in the E.U. for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR) and is currently under regulatory review in the U.S. and Canada. The approval is based on data from the NEURO-TTR study which was a Phase 3 randomised (2:1), double-blind, placebo-controlled, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. The 15-month study measured the effects of inotersen on neurological function and on quality-of-life by measuring the change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Inoterson provided significant benefit on both of these co-primary endpoints in the NEURO-TTR study, including improvement in disease relative to baseline measurements in both co-primary endpoints for a substantial portion of patients. Inotersen is associated with risk of thrombocytopenia and glomerulonephritis. Enhanced monitoring is required to support early detection and management of these identified risks. The most frequently observed adverse reactions during treatment with inotersen were events associated with injection site reactions. The other most commonly reported adverse reactions (over 10%) seen with inotersen were nausea, anaemia, headache, pyrexia, peripheral oedema, chills, vomiting, thrombocytopenia and platelet count decreased.
The approval is also based on data from the NEURO-TTR Open Label Extension (OLE) which is an ongoing study for patients who completed the NEURO-TTR study, designed to evaluate the long-term efficacy and safety of inotersen. For more information on inotersen, please visit www.TEGSEDI.eu.
ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS
hATTR amyloidosis is a progressive, systemic and fatal inherited disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, heart, intestinal tract, eyes, kidneys, central nervous system, thyroid and bone marrow.3,4 The progressive accumulation of TTR amyloid deposits in these tissues and organs leads to sensory, motor and autonomic dysfunction often having debilitating effects on multiple aspects of a patient’s life.4 Patients with hATTR amyloidosis often present with a mixed phenotype and experience overlapping symptoms of polyneuropathy and cardiomyopathy.4
Ultimately, hATTR amyloidosis results in death within three to fifteen years of symptom onset. Therapeutic options for the treatment of patients with hATTR amyloidosis are limited and there are currently no disease-modifying drugs approved for the disease. There are an estimated 50,000 patients with hATTR amyloidosis worldwide.4
References
1 European Medicines Agency. Tegsedi. Available here. Last accessed July 2018. 2 Benson, MD et al. (2018) Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis. NEJM. 379:22-31. 3 Damy T, et al. (2015) Cardiac Findings and Events Observed in an Open-Label Clinical Trial of Tafamidis in Patients with non-Val30Met and non-Val122Ile Hereditary Transthyretin Amyloidosis. J Cardiovasc Transl Res. 8(2):117-127. 4 Hawkins PN, et al. (2015) Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 47(8):625-638.
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