From the Concept to the Drug
The AOP Orphan team covers all essential aspects of product development, starting with preclinical research & toxicology, extending to pharmaceutical & clinical development, including regulatory affairs, quality management & pharmacovigilance.
Facts & Figures
AOP Orphan is a pharmaceutical company that invests a large part of its annual turnover in the research and development of new drugs. The company focuses on rare diseases, also known as orphan diseases. In Europe those are the diseases that occur in less than 5 per 10,000 residents.
As a fully integrated pharmaceutical company, AOP Orphan’s scope of activities includes research and development, as well as the production, marketing, and sales of drugs. AOP Orphan does not have its own laboratories, production facilities or clinics, but utilizes a worldwide network of professional service providers for the implementation of its research projects
"AOP Orphan has successfully developed a number of innovative treatment options in recent years. To arrive at these, our thinking is guided above all by patient needs."
"Our studies are more complex than for example diabetes or vaccine studies, since we ought to seek patients with rare diseases. Thus, the collaboration with doctors and specialized hospitals is of utmost importance."
"Conducting clinical trials in rare diseases is especially difficult, but also extremely rewarding, knowing that you provide a possible treatment option for these frequently overlooked patients."
Pipeline
The research team at AOP Orphan covers all essential aspects of product development, starting with preclinical research and extending to toxicology, pharmaceutical development, clinical development, regulatory affairs, quality management, pharmacovigilance, and project management. Experts in the individual specialities work as project managers in close cooperation with each other and with external service providers and partners, in order to implement the ambitious research projects of the organisation.
| Substance | Indication | Phase I | Phase II | Phase III | Registration | Marketed |
|---|---|---|---|---|---|---|
| Rapibloc® (Landiolol) | Tachycardia | |||||
| Marketed in EU | ||||||
| Tachycardia | ||||||
| Filing US 2020 | ||||||
| Septic shock | ||||||
| Phase IV in EU | ||||||
| BESREMi® (Ropeginterferon alfa-2b) |
Polycythaemia Vera | |||||
| EU Marketing Authorization 2/2019 | ||||||
| CML | ||||||
| Phase II | ||||||
| Prefibrotic myelofibrosis | ||||||
| Phase II | ||||||
| Anagrelide Prolonged Release |
Essential Thrombocythemia | |||||
| Filed EU 2016 | ||||||
| Nabilone | Pain medication | |||||
| Investigator Initiated Study, Phase II | ||||||
| AOP 2020 | Fragile X-Syndrome | |||||
| Phase II | ||||||
| Selisistat | Huntington's Disease | |||||
| Phase II | ||||||
Significance of the phases
Current Studies
Our research is conducted all over Europe and covers the entire spectrum of the various stages of development: from early clinical trials (phase I), extending to multinational approval studies in patients all over Europe (phase III), and including clinical trials with already approved products (phase IV). The ultimate goal of AOP Orphan research is always to find optimized solutions for the treatment of patients suffering from rare diseases.
LANDI-SEP
Study name: LANDI-SEP; EudraCT No. 2017-002138-22
Indication: patients with septic shock and persistent tachycardia
" LANDI-SEP is a Phase IV study in patients with septic shock, to control the heart rate of the patients with a short-acting beta-blocker. At present, we are at the early stage of our study, which is currently being conducted in Austria, Germany, Czech Republic and Italy."
- Phase IV, multicentre, prospective, randomised, open-label
- controlled study on Landiolol in patients with septic shock, admitted to an intensive care unit
Link to details on clinicaltrialsregister.eu
LANDI-PED
Study name: LANDI-PED; EudraCT No. 2015-001129-17
Indication: supraventricular tachycardia in pediatric patients
"The LANDI-PED study is investigating the efficacy and safety of Landiolol in children. This is very important and challenging because of the special characteristics of these young and often critically ill patients.”"
- A multicentre open-label study to investigate the effectiveness and safety of AOP Landiolol in controlling supraventricular tachycardia in paediatric patients
Link to details on clinicaltrialsregister.eu
CONTINUATION-PV
Study name: CONTINUATION-PV; EudraCT No. 2014-001357-17
Indication: Polycythaemia vera
"The CONTINUATION PV study is taking place in 12 countries, including France, Germany, Ukraine and Slovakia. The focus is on the long-term efficacy and safety of Ropeginterferon alfa 2-b."
- An open-label, multicentre, phase-IIIb study assessing the long-term efficacy and safety of AOP2014 and standard first line treatment (BAT) in patients with polycythaemia vera who previously participated in the PROUD-PV study
Link to details on clinicaltrialsregister.eu
Recent Publications
ROPEGINTERFERON ALFA-2B
Evidence for superior efficacy and disease modification after three years of prospective randomized controlled treatment of Polycythemia Vera patients with Ropeginterferon Alfa-2b vs. HU/BAT, Heinz Gisslinger et al. American Society Hematology ASH Annual Meeting, 2018, San Diego, oral presentation.
Heinz Gisslinger • et al. American Society Hematology ASH Annual Meeting 2018, oral presentation
Emmanuelle Verger et al. Blood, 8 -94, 2018.
http://www.bloodjournal.org/content/130/Suppl_1/320
Heinz Gisslinger • et al. American Society Hematology ASH Annual Meeting 2017, oral presentation
Jean-Jacques Kiladjian • et al. European Hematology Association EHA Annual Meeting 2017, oral presentation
http://www.bloodjournal.org/content/128/22/476
Heinz Gisslinger • et al. American Society Hematology ASH Annual Meeting 2016, oral presentation
Nicole Them • et al. American Journal of Hematology, 90(4):288-94, 2015
ANAGRELIDE RETARD
Heinz Gisslinger et al. British Journal of Haematology, 2019.
Petro E. Petrides • et al. Clinical Pharmacology in Drug Development, 7 (2):129 -131, 2018
http://www.bloodjournal.org/content/128/22/475?sso-checked=true
Heinz Gisslinger • et al. American Society Hematology ASH Annual Meeting 2016, oral presentation
Heinz Gisslinger • et al. European School of Hematology EHA 21st Congress, Jun 9-12, 2016
LANDIOLOL
Unger, M., et al. (2018); Trials 19(1): 637.
Günther Krumpl • et al. J Cardiovasc Pharmacol. 71(3): 137-146, 2018
Günther Krumpl • et al. J Cardiovasc Pharmacol. 70(1):42-51, 2017
Günther Krumpl • et al. Eur J Clin Pharmacol. 73(4):417-428, 2017
Günther Krumpl • et al. Eur J Pharm Sci. 92:64-73, 2016
"To provide patients with effective drugs, COLLABORATION is the key to success – both within and between the pharmaceutical industry and academia."
"To continue to make a valuable contribution to medical research in rare diseases, as it is our core competence."
"A trustful relationship with investigators and study staff in every single center, quick communication, competence synergies – all of these aspects are of vital importance for the success of our studies."