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CTEPH affects more than 1,000 people in Spain1

  • After 25 years focused on research into rare diseases with complex management, the Austrian pharmaceutical company AOP Orphan has arrived in Spain to continue offering therapeutic options to these patients.
  • Chronic thromboembolic pulmonary hypertension is a rare disease characterised by blood clots blocking the pulmonary arteries.
  • The new product, subcutaneous treprostinil, is indicated for the treatment of CTEPH in adult patients in WHO functional class III or IV and: with inoperable, persistent or recurrent CTEPH after surgical treatment.
  • Specifically, this drug is the only prostacyclin approved for the treatment of patients with inoperable CTEPH and the only drug therapy for patients in WHO functional class IV.

Madrid, October 6th, 2021. After 25 years focused on research into rare diseases with complex management, the Austrian pharmaceutical company AOP Orphan has just arrived in Spain. Based in Vienna, the company has more than 400 employees in 20 countries and is pioneer in offering innovative therapeutic solutions to patients with rare diseases "Since our inception, we have had a very clear mission: to transform the daily lives of people with these pathologies through a deep understanding of the mechanisms of the disease, the specific needs of patients and by working closely with the healthcare professionals involved in their treatment," says Marco Messina, Managing Director of AOP Orphan in Spain. Specifically, the company develops and distributes treatments in four therapeutic areas: haemato-oncology, cardiology and pneumology, neurology and metabolic disorders, and intensive care medicine. "The aim is to respond to the individual needs of these patients and to offer them therapeutic options that contribute to improving their lives," he adds. More than 80 % of AOP Orphan´s therapies are produced in Europe.

In this regard, the commitment to innovation since the creation of the company highlights particularly: "For more than 25 years, the company has focused on R&D to drive a real change in the quality of life of these patients, being a pioneer in the field of rare diseases. In fact, for some of these pathologies, AOP Orphan is the only provider of therapeutic solutions worldwide. Moreover, 20% of AOP Orphan's turnover is reinvested in R&D," says Uriel Garcia Ibarz, Medical Director of AOP Orphan in Spain.

AOP Orphan's arrival in Spain is the result of the company's expansion and internationalisation and coincides with the launch in our country of subcutaneous treprostinil. It is a new drug indicated for the treatment of CTEPH in adult patients in WHO functional class III (patients who are symptom-free at rest but whose daily activities are limited by shortness of breath or fatigue) or IV (patients who are symptomatic at rest and have severe symptoms when performing any physical activity) and: with inoperable, persistent or recurrent CTEPH after surgical treatment. 

This is currently the only drug therapy for functional class IV patients who cannot tolerate soluble guanylate cyclase stimulator or need combination therapy. In addition, is the only prostacyclin approved for the treatment of non-operable CTEPH patients.

Along this treatment, AOP Orphan has already launched two other products in Spain and continues to work to expand its portfolio in our country. These are: treprostinil for the treatment of pulmonary arterial hypertension (PAH) and argipressin for the treatment of catecholamine-refractory hypotension associated with septic shock in patients over 18 years of age. In addition, as of January 2021, the AEMPS - through the Special Use Medicines Platform (MSE) - has authorised ropeginterferon alfa-2b as monotherapy in adults for the treatment of polycythaemia vera without symptomatic splenomegaly.

What is CTEPH and how can this new launch contribute to its treatment?

Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare disease that, similarly to other types of pulmonary hypertension (PH), reduces blood flow and increases pressure in the blood vessels of the lung2. The most common symptoms3 are dyspnoea, dizziness, tiredness, fainting, palpitations or abnormal heartbeat, dry cough, chest pain, swollen ankles or legs. , The current prevalence of CTEPH in Spain has increased to 22.5 cases/million inhabitants, so it is estimated that it affects more than 1,000 people in Spain. 

Besides, CTEPH is a severe condition that needs special care and frequently a combination of treatments: pulmonary endarterectomy, balloon pulmonary angioplasty and medical treatments. However, in an international registry of 679 newly diagnosed CTEPH patients, 36% of them (247) were classified as inoperable4. “The approach to CTEPH has evolved significantly in the last decades and, currently, patients who have for example chronic total oclusions and are inoperable can be treated with medical treatments. They are very valid alternatives and also could improve outcomes after mechanical treatments”, says Dr. Irene Lang, clinical cardiologist and professor of vascular biology at the Medical University of Vienna. “By my experience, subcutaneous treprostinil is a safe and well tolerated treatment that should constantly monitored by healthcare professionals experienced in treating this type of pulmonary hypertension. So, it is not only a drug but a entire group of caregivers plus experience and support”, adds Dr. Lang.

This new treatment acts in a similar way to prostacyclin, a natural substance that widens blood vessels and prevents platelets (blood components) from sticking together to form clots. In patients with CTEPH5, these effects of subcutaneous treprostinil prevent clots from forming and reduce blood pressure in the pulmonary artery, thus improving the symptoms of the disease.
In addition, the main study conducted on this drug (called CTREPH) has shown that it significantly improves physical capacity in patients with CTEPH. In particular, long-term subcutaneous treprostinil is shown to be safe and effective, leading to improvements in distance (as determined by the 6-minute walk test), haemodynamics, WHO functional class and NT-proBNP (myocardial stress marker) levels in these critically ill patients.

This drug is administered subcutaneously and is available in concentrations of 1, 2.5, 5 mg/ml and 10 mg/ml unlike other therapeutic options that offer a maximum daily dose.

About chronic thromboembolic pulmonary hypertension (CTEPH)

Chronic thromboembolic pulmonary hypertension (CTEPH) is a rare disease that, similarly to other types of pulmonary hypertension (PH), reduces blood flow and increases pressure in the blood vessels of the lungii. ‘Thromboembolic’ refers to the complete or partial blockage of a blood vessel by a blood clot that has broken away from where it was formed. In Spain, CTEPH prevalence increased to 22.5 per million population, so it is estimated that there are more than 1,000 patients in Spain.
 

About AOP Orphan

AOP Orphan Pharmaceuticals GmbH is an international pharmaceutical company focused on rare and special diseases. Headquartered in Vienna, Austria, with subsidiaries and representative offices across Europe, the Middle East , the company has become an established provider of integrated therapy solutions over the past 25 years. This development has been made possible by a continually high level of investment in research and development on the one hand and a highly consistent and pragmatic orientation towards the needs of all its stakeholders on the other - especially patients and their families, but also the doctors and care professionals treating them. In the third quarter of 2020, AOP Orphan took over Amomed Pharma GmbH and SciPharm s.r.l., two European health care companies, continuing its consistent path of growth into a pan-European health care group specializing in rare and special diseases.
 

Contact for the media: 

Omnicom PR Group agency
Sara Portillo / Pilar Piqueras
Sara.portillo@omnicomprgroup.com; pilar.piqueras@omnicomprgroup.com 
Tel. 91 91 42 700 / 91 91 42 845

1 https://www.revespcardiol.org/es-videos-hipertension-pulmonar-tromboembolica-cronica-espana--242

2  Lang IM and Madani M. Update on chronic thromboembolic pulmonary hypertension. Circulation. 2014;130:508-18.

 3 https://cima.aemps.es/cima/dochtml/p/1191419002/P_1191419002.html

 4 Pepke-Zaba J et al. Circulation 2011;124:1973–81

5  https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(18)30367-9/fulltext