Targeting Rare Diseases
and Critical Care
AOP Health is the European pioneer for integrated therapies for rare diseases and in critical care.
As a privately owned company AOP Health is devoted to long-term commitment, high quality, and continuity. For a small number of very special diseases the company is the sole supplier of some key therapeutic agents throughout the world.
Why did you found AOP Health?
“When founding AOP Health in 1996 – as AOP Orphan – my aim was to make targeted treatment available to the maximum number of patients with rare diseases. I realized that these patients are frequently left alone in their suffering. As an entrepreneur the complex environment of rare diseases was a great challenge, but also an opportunity to be successful in this niche.”
What role did AOP Health’s sense of entrepreneurial responsibility play in the founding of the company?
“Entrepreneurial responsibility is no additionally imposed obligation for AOP Health, but the attitude that played an indispensable role at the very time the company was founded. AOP Health’s objective to provide effective help for persons with rare diseases originated from this sense of responsibility and has made the company what it is today.“
AOP Orphan – now AOP Health – was founded in 1996 by Dr. Rudolf Widmann with the aim of developing and providing therapy options for patients with rare diseases, and then introducing the therapy options first in Austria, the European core countries of AOP Health, and finally in the international market.
“I worked for several years in large international pharmaceutical companies and observed that there were some therapy options for rare diseases, but they did not reach the large majority of the patients.”
Facts & Figures
Over the past 25 years, the group has become an established provider of integrated therapy solutions from its headquarters in Vienna, its subsidiaries and representative offices throughout Europe and the Middle East, as well as through partners worldwide. Since the very start the company has been focused on the individual needs and treatments of patients. Based on its research-centered approach, AOP Health has a profound understanding of rare diseases and critical care. Therefore, the company has been able to position itself as an innovative front-runner in the development of effective therapies and drugs.
AOP Health, with over 20 subsidiaries and representative offices, now operates
in all European countries as well as in the United Arab Emirates and Israel. Furthermore, the company is active internationally through its strategic partnerships.
Vision & Mission
History of more than 25 years means many significant milestones — in research, social, business spheres and regulatory affairs — that were and are important for AOP Health.
2022 – New brand name: AOP Health
The brand name was changed to AOP Health to reflect the expansion of business activities beyond rare diseases covering all of the Group’s business segments including critical care.
AOP Orphan Group
AOP Orphan took over the Viennese pharmaceutical company Amomed and the Luxembourgish health-tech company SciPharm.
On Feb 15th EMA approves AOP Orphan’s BESREMi® as firt-line monotherapy in adults for the treatment of Polycytheamia vera (PV) without symptomatic splenomegaly.
Treprostinil and PAH
The paper “Subcutaneous treprostinil for the treatment of severe non-operable chronic thromboembolic pulmonary hypertension (CTREPH): a double-blind, phase 3, randomised controlled trial" was published by R. Sadushi-Kolici R; Jansa P; Kopec G; Torbicki A; Skoro-Sajer N; Campean IA; Halank M; Simkova I; Karlocai K; Steringer-Mascherbauer R; Samarzija M; Salobir B; Klepetko W; Lindner J; Lang IM (Source: https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(18)30367-9/fulltext). The authors conclude that sc. Treprostinil significantly improves the cardiovascular situation and performances figure of patients with CTREPH.
Landiolol, a new ultra-high selective short acting beta 1 adrenoceptor blocker, developed by AOP in 2 forms (20 mg/2ml concentrate, 300 mg powder) is registered in several European countires for the treatment of non-compensatory sinus tachycardia and tachycardic supraventricular arrhythmias. The drug uses a new AOP Orphan-developed dosing algorithm to facilitate the administration and proves to be superior to Esmolol in its ability to reduce heart rate more than blood pressure.
Pitolisant, also known as tiprolisant, is a potent and selective inverse agonist of the histamine H3 receptor (Ki = 0.16 nM), and was approved for the treatment of narcolepsy in 2016. Pitolisant was developed by Jean-Charles Schwartz, Walter Schunack, and colleagues, after the former discovered the H3 receptor. It was the first H3 receptor inverse agonist to be tested in humans or introduced for clinical use.
Beta-blocker for severe sepsis
The paper “Beta-blocker use in severe sepsis and septic shock: a systematic review.” by Sanfilippo F1, Santonocito C2, Morelli A3, Foex P4 describes the ability of beta blockade to modulate sepsis-induced alterations at the cardiovascular, metabolic, immunologic and coagulation levels.
Start of the PROUD PV Study
The PROUD-PV study is a phase III study to compare the efficacy and safety of the novel ropeginterferon α-2b versus hydroxyurea in both HU-naive and currently treated patients, diagnosed with Polycythemia vera.
Nabilone Approval in Austria
Nabilone is a synthetically produced substance belonging to a group of compounds known as cannabionoids, used for the treatment of CINV.
Tetrabenazine Approval in Central Europe
Tetrabenazine approval for the treatment of Huntington's chorea in 23 European countires till the end of 2011.
In 2010 EMA designated UDCA an "orphan medicine" status — a medicine used for the treatment of primary biliary cholangitis.
Renaissance of Interferon (IFNα)
IFNα has been used to treat malignant and viral disorders for more than 25 years.
A few clinical trials starting from 2008 showed that interferon is effective in normalizing blood counts, reducing the mutant JAK2 allele burden and, in some cases, reversing disease progression.
The Janus Kinase 2 gene (JAK2) was discovered in 2005. It provides instructions to cells for making the JAK2 protein. The test looks for mutations in JAK2 that are associated with bone marrow disorders caused by the production of too many blood cells.
Prostacyclin Analogues Approval
Subcutaneous prostacyclin analogue approved for the treatment of pulmonary arterial hypertension (PAH) in Europe.
European Union Expansion
The European Union (EU) has expanded several times throughout its history via the accession of new member states. The EU expansion towards Eastern and Central Europe and the Western Balkans had a positive influence on the territorial expansion of AOP Orphan as well, creating a basis for providing treatments for rare diseases in all AOP Orphan core markets.
1st Approval of Anagrelide in Europe
First marketing authorization for Anagrelide in Austria for treatment of patients with essential thrombocythemia.
The Orphan Regulation EC no 141/2000
European Parliament adopted Regulation EC No 141/2000 The Orphan Regulation of Dec 16th, 1999. The Regulation lays down the EU procedure for the designation of orphan medicines, defines incentives for the development and placing of designated orphan medicines in the market, and establishes the Committee for Orphan Medicinal Products (COMP).
Foundation of AOP Orphan in Vienna
Dr. Rudolf Widmann established a successful innovative pharmaceutical company focussed on the treatment of rare diseases in 1996 in Vienna, Austria.
Since its very start AOP Health has borne the corporate responsibility of further life quality improvement for patients with severe, rare, life-threatening diseases as well as patients in critical conditions. We at AOP Health conduct intensive research for this purpose, believing that developing and providing solutions for patients is the most important part of the AOP Health corporate responsibility. When it comes to wanting the best for the patients, we stand side-by-side with each other as a team, with researchers, physicians, therapists, caregivers and
, patient organizations.
AOP Health is keen to keep one of its greatest assets – it is closely networked with the key specialists in all markets and is therefore able to help patients with rare diseases from Austria throughout the world.
From its inception AOP Health's culture is shaped by the entrepreneurial spirit of its founder. We are proud to be a company that encourages employees to come up with new ideas and to show ownership and initiative every day. To help patients with rare diseases and in critical care is our key motivator. Through close interaction with the scientific community, care givers and partners we aim to provide therapies and help patients beyond just providing drugs.
Thanks to our advancements in the field of therapies, an increasing number of patients survive their disease or are able to live with it for a longer period of time. In the next few decades, personalized medicine, individual treatment opportunities will emerge more and more as the standard in AOP Health’s therapy concepts as well.
Novel technologies such as next-generation sequencing (NGS) will play an equally crucial role as the increasing quantities of big data and conclusions derived from the daily lives of patients also beyond the strictly regulated conditions of clinical trials, e.g. real-world evidence.
Furthermore, the increasing health competence, health literacy of patients and their family members will also determine the future of pharma and health care sector.