Cancer is one of the leading causes of death worldwide (1) and e.g. in Germany the second leading cause of death after cardiovascular diseases (2).
Decades of intensive research in oncology have provided a variety of therapies for numerous cancer indications. Therefore, toady more than half of the affected patients in Germany can hope for permanent cure (2). However, for a variety of cancer disease new therapeutic options need to be developed.
With this, a special role comes to rare or ultra-rare diseases (this reflects e.g. in the EU disease with less than 230.000 patients per year or five per 10.000 inhabitants (3)). Major pharmaceutical companies focus under usual market conditions often on oncological indications with bigger patient numbers. Therefore therapies for patients with rare diseases are only rarely developed. This is why these drugs are called orphan drugs.
To cover this situation, legal frameworks were implemented to encourage and support the development of orphan drugs such as the Orphan Drug Act in the US in 1983 and the EC Regulation on orphan medicinal products in 2000 in the EU. These circumstances allow also smaller companies like Oncoscience AG to develop orphan drugs effectively and successfully. Currently Oncoscience AG, as part of Innokeys Ptd Ltd, accomplishes the clinical development of Nimotuzumab in Europe. Nimotuzumab is a monoclonal antibody that can be used for the treatment of several cancer diseases. Nimotuzumab has Orphan Drug Status in both the EU and the US for the indications diffuse intrinsic pontine glioma and pancreatic cancer – both indications where Oncoscience AG conducts research.
(3) REGULATION (EC) No 141/2000