SMC Accepts Adempas® (riociguat) to Treat Rare Respiratory Disease in Scotland

The Scottish Medicines Consortium (SMC) has accepted Adempas for restricted use in the treatment of adult patients with 'inoperable CTEPH'1

8th December 2014 – Bayer HealthCare today welcomes the announcement from the Scottish Medicines Consortium (SMC) that the first specifically licensed treatment for chronic thromboembolic pulmonary hypertension (CTEPH) - Adempas® (riociguat) - will be made available for restricted use in Scotland.1

Adempas is accepted by the SMC for the treatment of adult patients with World Health Organisation (WHO) functional class II to III* with "…inoperable CTEPH, persistent or recurrent CTEPH after surgical treatment and to improve exercise capacity for patients in whom a PDE5 inhibitor is inappropriate, not tolerated, or ineffective. It is restricted to prescribing by specialists in the Scottish Pulmonary Vascular Unit."1

CTEPH causes severe narrowing of blood vessels in the lungs leading to high blood pressure in the pulmonary arteries (leading from the heart to the lungs) - giving rise to 'pulmonary hypertension' (PH). The heart has to work very hard to push blood through the narrowed blood vessels to be infused with oxygen in the lungs. The resulting symptoms can include breathlessness and fatigue upon minimal physical activity, chest pain and dizziness. Untreated, the pulmonary hypertension associated with CTEPH progressively worsens and eventually leads to heart failure and premature death.2,3

Adempas is the first licensed treatment to stimulate soluble guanylate cyclase (sGC) - an enzyme that catalyses the synthesis of a signalling molecule called cyclic guanosine monophosphate (cGMP).

Within cells cGMP plays an important role in regulating processes that influence vascular tone, proliferation of cells, fibrosis and inflammation.4

The SMC Advice states that, following a full submission considered under the end of life/orphan medicine process: "Riociguat demonstrated significant improvement compared with placebo in exercise capacity, in terms of 6-minute walk distance, in patients with inoperable CTEPH or persistent or recurrent pulmonary hypertension after pulmonary endarterectomy." 1

Pulmonary endarterectomy is the standard and potentially curative treatment for CTEPH. It is a highly specialised surgical procedure in which the blood vessels of the lungs are cleared of diseased scar tissue that develops from accumulated blood clots.2 However, CTEPH is inoperable in a substantial proportion of patients5 and up to 10-15% of patients who undergo pulmonary endarterectomy have persistent or recurrent PH following surgery.6 Riociguat can now be considered in these two groups of patients.1

Dr Martin Johnson, Consultant Physician at Scotland's only specialist pulmonary vascular unit located at the Golden Jubilee National Hospital in Glasgow, commented: "We are very excited to have access to a proven medical treatment for patients with CTEPH. The data from the clinical studies are compelling and our own experience of this medication through the pre-licensing trials has been very positive. We are especially pleased that Scotland is the first area within the UK to have access to this medication."4,7

"Ongoing commitment towards research and making new treatments available for rare and complex diseases like CTEPH is so important to those who suffer from them. So we are very positive about the SMC's decision to accept riociguat for restricted use in Scotland," said Dr Iain Armstrong, Chairman of the Pulmonary Hypertension Association UK.

*The WHO classification reflects the severity of the disease on physical activity; Class I (no limitation in physical activity) Class II (some limitation), Class III (severe limitation) and Class IV (symptoms at rest and limited physical activity).7

-Ends-


For further information, please contact:
Hayley Knight Tel: +44 1635 563523
Bayer HealthCare Mob: +44 7788 365833
E-mail: [email protected]

Notes to Editors:

About Scottish Medicines Consortium (SMC)
The SMC reviews and accepts for use those newly licensed drugs that clearly represent good value for money to NHSScotland. SMC works to make sure that those drugs are accepted for routine use as quickly as possible so that they can benefit patients. Once the SMC has issued guidance, which it aims to do as soon as possible after the medicine comes onto the market, Area Drug and Therapeutic Committees in each of Scotland's 14 health boards then examine the recommendations to approve use locally.8

More information about SMC can be found at www.scottishmedicines.org.uk

CTEPH Prevalence
The UK National Audit on Pulmonary Hypertension for 2012 showed the estimated prevalence of CTEPH in Scotland to be 16.1 cases per million population and 18.9 cases per million population in England.9

CTEPH may evolve after prior episodes of acute pulmonary embolism but the link is not yet completely understood. Although evidence is not definitive, most experts believe that the incidence of CTEPH after acute pulmonary embolism is 0.5–2%.7

About Pulmonary Hypertension
Pulmonary hypertension, it is a serious and progressive condition of the heart and lungs in which the blood pressure in the pulmonary arteries is above normal. It is characterised by increasing limitations on physical activity, right heart failure and premature death.3

There are different types and five classifications of pulmonary hypertension based on the underlying cause of disease, each classification requiring a specific treatment approach. CTEPH is one of these disease classifications. Correct diagnosis and exact classification of disease type are important as they will determine the proper treatment approach.3

The most common symptoms of pulmonary hypertension include shortness of breath, fatigue, dizziness and fainting, all of which are worsened by exertion. As some of these symptoms can easily be mistaken for other conditions, diagnosis can be delayed by months or years.3 Continuous treatment monitoring is vital to ensure that patients receive optimal care for their particular type and stage of disease.7

Pulmonary hypertension only affects around 6,000 people in the UK each year.9

More information about CTEPH and pulmonary hypertension can be found at The Pulmonary Hypertension Association website www.phassociation.org


About riociguat
Riociguat was licensed for use in the EU under the name Adempas® for use in CTEPH and pulmonary arterial hypertension (another classification of pulmonary hypertension) in March 2014. The drug has an 'orphan designation' which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.10

Riociguat, the first of a novel class of compounds, is a daily oral treatment that stimulates an enzyme called soluble guanylate cyclase in the blood vessels of the lungs. This causes the blood vessels to relax and widen. This helps to lower the blood pressure in the lungs and improve symptoms of CTEPH.4,10

The two-year results of the riociguat open-label extension study CHEST-2 confirm that the improvements in exercise capacity and WHO Functional Class as observed in the pivotal Phase III study CHEST-111 were sustained.12 Exercise capacity as measured by the 6-Minute Walk Distance (6MWD) test is a marker of disease severity and a predictor of survival in patients suffering from pulmonary hypertension.13

More details about riociguat can be found at the European Medicines Agency website: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002737/human_med_001733.jsp&mid=WC0b01ac058001d124

For the full list of all side effects reported with riociguat and the full list of restrictions of use, see the full Summary of Product Characteristics. This can be accessed by entering the drug name in the search box of the electronic Medicines Compendium (eMC) website www.medicines.org.uk

Riociguat is made available to Scotland under a Patient Access Scheme (PAS), which improves the affordability of treatment.1

About Bayer HealthCare
The Bayer Group is a global enterprise with core competencies in the fields of health care, agriculture and high-tech materials. Bayer HealthCare, a subgroup of Bayer AG with annual sales of EUR 18.9 billion (2013), is one of the world's leading, innovative companies in the healthcare and medical products industry and is based in Leverkusen, Germany. The company combines the global activities of the Animal Health, Consumer Care, Medical Care and Pharmaceuticals divisions. Bayer HealthCare's aim is to discover, develop, manufacture and market products that will improve human and animal health worldwide. Bayer HealthCare has a global workforce of 56,000 employees (Dec 31, 2013) and is represented in more than 100 countries.
Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.


References
1. Scottish Medicines Consortium. Riociguat (Adempas) Advice. Available at: www.scottishmedicines.org.uk Last accessed: December 2014.

2. McNeil K and Dunning J. Chronic thromoembolic pulmonary hypertension (CTEPH), Heart 2007;93(9):1152–1158.

3. Rosenkranz S. Pulmonary hypertension: current diagnosis and treatment. Clin Res Cardiol 2007;96(8):527-541.

4. Adempas® Summary of Product Characteristics. Available at https://www.medicines.org.uk/emc/medicine/28739 Last accessed: November 2014.

5. Condliff R et al. Improved outcomes in medically and surgically treated chronic thromoembolic pulmonary hypertension. Am J Respir Crit Care Med. 2008;177(10):1122-7.

6. Rahnavardi M et al. Pulmonary thromboendarterectomy for chronic thromboembolic pulmonary hypertension: a systematic review. Ann Thorac Cardiovasc Surg. 2011;17(5):435–445.

7. Galie N et al. Guidelines for the diagnosis and treatment of pulmonary hypertension: The Task Force for the Diagnosis and Treatment of Pulmonary Hypertension of the European Society of Cardiology (ESC) and the European Respiratory Society (ERS), endorsed by the International Society of Heart and Lung Transplantation (ISHLT). Eur Heart J 2009;30(20):2493-2537.European Medicines Agency.

8. Scottish Medicines Consortium website. Available at www.scottishmedicines.org.uk Last accessed: November 2014.

9. Health and Social Care Information Centre. Part of the Government Statistical Service. National Audit of Pulmonary Hypertension 2013 Report for the audit period April 2012 to March 2013. Available at: http://www.hscic.gov.uk/catalogue/PUB13318/nati-pulm-hype-audi-2013-rep.pdf Last accessed: November 2014.

10. The European Medicines Agency (EMA) Adempas (riociguat) European Public Assessment. March 2014. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002737/human_med_001733.jsp&mid=WC0b01ac058001d124 Last accessed: November 2014.

11. Ghofrani H, et al. Riociguat for the treatment of chronic thromboembolic pulmonary hypertension. N Engl J Med 2013;369(4):319-329.

12. Simonneau G, et al. Riociguat for the treatment of chronic thromboembolic pulmonary hypertension: 2-year results from the CHEST-2 long-term extension. European Respiratory Society International Congress; 6–10 September 2014; Munich, Germany [P1802].

13. Miyamoto S, et al. Clinical correlates and prognostic significance of six-minute walk test in patients with primary pulmonary hypertension. Comparison with cardiopulmonary exercise testing. Am J Respir Crit Care Med 2000;161:487–492.

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