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At AOP Orphan are European pioneers in the field of Orphan Diseases. We research, develop, produce, and distribute innovative solutions for Rare Diseases worldwide.

Dr. Rudolph Widmann established AOP Orphan in Vienna in 1996 in order to provide individualized treatments for patients suffering from Rare Diseases. AOP Orphan has especially focused in the areas of hematology and oncology, cardiology, pulmonology, neurology, psychiatry and metabolic diseases. 

Thanks to 15 years of experience in complex individualized treatments, AOP Orphan has obtained exceptional competence as well as market presence in the field of Orphan Diseases. The fact that AOP Orphan is privately owned assures a long-term engagement in the market along with high quality services and products. AOP Orphan is also the only provider of key therapies for certain special indications.

AOP Orphan experts work closely at an international level with leading health care professionals. This has given them the ability to react quickly towards needs and developments and to push research and development projects forward.  When implementing these projects, AOP Orphan has a worldwide network of highly qualified partners that it can reach out to. These strategical alliances make sure that AOP Orphan as a middle-sized enterprise is able to compete with top-pharmaceutical companies. 

AOP Orphan is currently one of the leading suppliers in the pharmaceutical industry, and as an international Austrian business, AOP Orphan generates sales of more than 50 million Euros. The company has an export quota of around 70% and over 100 employees. AOP Orphan’s branch offices are situated within its core markets in the EU, Switzerland, and the Middle East. Overall AOP Orphan has offices in 12 different regions.  The international markets are handled and operated by close partners of AOP Orphan. 

The company is led by the founder Dr. Rudolph Widmann, as well as Dr. Georg Fischer, Dr. Christoph Klade, Mag. Karin Pernkopf, Ms. Elfriede Gumplmayr and Douglas Lloyd-Fell. 

Area of Treatment

Hematology & Oncology

In the branch of Hematology and Oncology, AOP Orphan focuses on myeloproliferative disorders—a group of malignant hematologic disorders that lead to the degeneration of the hematopoetic cells in the myeloid cell line.

  • Essential Thrombocythemia—a Rare Disease of the hematopoetic system in the bone marrow that causes an overproduction of platelets (thrombocytes) in the blood.
  • Polycythemia Vera—in this case there is a progressive increase in the production of erythrocytes (red blood cells). Most often an increase in the leukocytes and thrombocytes occurs as well.
  • Primary Myelofibrosis—pri myelofibrosis develops into a progressive scarring of the bone marrow,  which is often accompanied by anemia.
  • Chronic Myelogenous Leukemia (CML)—CML is a malignant blood disease that has a slow progression rate.  It is caused by the clonal expansion of diseased blood stem cells which cause the overproduction of white blood cells (leukocytes).

Cardiology & Pulmonology

In the branch of cardiovascular diseases, AOP Orphan has specialized in two indications:

  • Cardiac Arrhythmias—are harmless, entirely normal short-term changes in the heartbeat, which can turn into a life-threatening situation when suffering from preconditions such as a pre-damaged heart muscles in need of acute treatment.
  • Pulmonary Arterial Hypertension (PAH): a disease that is characterized by increased pressure in the pulmonary arteries that leads to an escalating strain to the right ventricular. Untreated it causes a chronic over-exertion on the right side of the heart and in the end leads to heart failure and potential death. Early diagnosis and adequate therapy are of the highest importance for the prognosis and quality of life of the patient.

Neurology and Psychiatry

There are a small number of diseases located in the neurology region that are also considered Rare Diseases. AOP Orphan is focused on the diseases listed below:

  • Huntington’s Disease: a hereditary neurology disorder of the central nervous system that leads to a progressive degeneration of the brain cells.  As a result motor disorders, cognitive disorder and behavioral disorders occur.
  • Multiple Sclerosis (MS)—an inflammatory disease of the nervous system. It is the most common neurological disease and it is usually diagnosed between the age of 20 and 40.
  • Rolando’s Epilepsy—a benign type of epilepsy that appears often without any recognizable preliminary damage during childhood. It is distinguished by the type of epilepsy episode and whether the EEG exhibits characteristic changes.  The prognosis is favorable.  Rolando’s Epilepsy belongs to the most common form of childhood seizures.

Metabolic Disorders

In the branch of Metabolic Disorders, AOP Orphan focuses on those areas where there is a genetically determined or acquired enzyme deficit that triggers the disease— as is the case with Cystic Fibrosis (Mucoviscidosis).

  • Cystic Fibrosis (Mucoviscidosis) is a congenital metabolic disorder characterized by frequent lung infections, chronic coughing, failure to thrive, and salty sweat. Mucoviscidosis is one of the most common genetically determined metabolic disorder in the group of Rare Diseases.


AOP Orphan Pharmaceuticals AG
Mag. Daniela Gruber
Wilhelminenstraße 91/IIf
1160 Wien
T +43 1 503 72 44-42
F +43 1 503 72 44-61